Researchers tested delgocitinib cream in 98 adolescents with moderate to severe hand eczema. After 16 weeks, 64% of teens using the cream had clear or almost clear skin, compared to 29% using a placebo cream. The treatment was well tolerated with no serious side effects.
The Lancet. Child & adolescent health · NCT05355818
Researchers tested tavapadon, a once-daily pill, in 304 people with early Parkinson's disease. After 26 weeks, those taking tavapadon had significantly better movement and daily function scores compared to those on a sugar pill, with side effects mainly including nausea, headache, and dizziness.
The Lancet. Neurology · NCT04223193
Researchers tested an 8-week online program combining talk therapy and gentle exercise for teens with ongoing sickle cell pain. Over 4 in 5 teens reported less pain after completing the program, and the improvement lasted at least 3 months.
Blood advances · NCT06110754
Researchers tested a targeted combination therapy called venetoclax-obinutuzumab against traditional chemotherapy in people newly diagnosed with chronic lymphocytic leukemia. More patients on the targeted therapy had no detectable cancer in their blood after treatment.
Blood · NCT04285567
Scientists tested an online therapy program called ReSeT for children who developed stress symptoms after being hospitalized for injuries like broken bones or burns. Kids who used the program had fewer stress symptoms than those who got usual care, and the benefits lasted at least six months.
JAMA pediatrics · NCT04838977
The RUBY trial tested reni-cel, a one-time gene editing treatment for patients with severe sickle cell disease who experience frequent painful crises. Researchers modified each patient's own stem cells to reactivate fetal hemoglobin — preventing cells from taking the sickle shape that causes blockages and organ damage.
New England Journal of Medicine · NCT04853576
Military personnel with PTSD received either real or sham magnetic brain stimulation plus intensive therapy. Those who got real stimulation had greater symptom relief that lasted longer—85% still improved at one month versus 59% with sham treatment.
JAMA network open · NCT02853032
This study tested bimekizumab, an injection for moderate to severe plaque psoriasis, against secukinumab (another approved treatment). After one year, 62% of people on bimekizumab had completely clear skin and minimal impact on quality of life, compared to 43% on secukinumab.
JAMA dermatology · NCT03536884
TEMPO-3 tested tavapadon — a once-daily selective dopamine pill — added to levodopa in 507 people with Parkinson's and motor fluctuations. Tavapadon added about 1.1 hours of good on-time per day.
JAMA Neurology · NCT04542499
OCULUS tested whether holding one weekly or daily dose of a GLP-1 drug (like Ozempic or Mounjaro) before upper endoscopy reduces stomach contents that complicate sedation. The trial was stopped early — 25% who continued had retained food vs. 3% who held the dose.
JAMA Internal Medicine · NCT06533527
A daily targeted pill reduced disease progression risk by 84% in unresectable Stage III EGFR-mutated non-small cell lung cancer after chemoradiation — a population with few good options after completing standard treatment.
New England Journal of Medicine · NCT03041701
Researchers tested a higher dose of nusinersen in babies and children with spinal muscular atrophy. After six months, babies who received the higher dose showed major improvements in motor function, while those who didn't receive treatment got worse.
Nature medicine · NCT04089566
STRENGTH tested intrathecal onasemnogene abeparvovec — a spinal-fluid version of Zolgensma — in 27 children and teens with SMA who had stopped nusinersen or risdiplam. The 52-week safety profile matched what was seen in younger, untreated patients.
Nature Medicine · NCT05386680
Researchers tested a one-time gene transfer therapy given through the spine in children with spinal muscular atrophy who could sit but couldn't walk. After one year, children who received the treatment showed significantly better muscle function compared to those who didn't.
Nature medicine · NCT05089656
Researchers tested an online self-help program to help people stop taking benzodiazepines after long-term use. People who used the digital tool were over 5 times more likely to completely stop their benzodiazepine compared to those who received usual care from their doctors.
JAMA network open · NCT04572750
Adding abemaciclib to hormone therapy after surgery cut the risk of cancer returning by 35% in high-risk hormone receptor-positive breast cancer patients — a meaningful improvement over hormone therapy alone.
Journal of Clinical Oncology · NCT02326324
This 37-patient trial gave a personalized T cell therapy targeting five tumor proteins to people with advanced pancreatic cancer. In patients responding to chemotherapy, the disease control rate was 85%; two of nine patients with resectable disease were still cancer-free 5+ years later.
Nature Medicine · NCT03192462
This analysis of the 1,230-person SUMMIT trial looked at suicide thoughts during a brief 6-8 session behavioral activation therapy for pregnant and postpartum people with depression. Suicide thoughts dropped 25% with each added session and 80% by 3 months.
JAMA Psychiatry · NCT04153864
This study integrated 137 patients across five trials of large B-cell lymphoma to test whether ultra-sensitive tumor DNA testing predicts relapse better than standard PET scans. At end of treatment, undetectable tumor DNA predicted a 97% chance of remaining cancer-free at 2 years.
Journal of Clinical Oncology · NCT04134936
Researchers tested whether people with migraine could reduce their disability by using a smartphone app that taught progressive muscle relaxation—a technique where you tense and relax different muscle groups. After three months, those who used the app had significantly less migraine-related disability than those who only tracked their symptoms.
JAMA network open · NCT04281030
Researchers tested orforglipron, a pill taken once daily, in adults with type 2 diabetes managed with diet and exercise. After 40 weeks, people taking the medication lowered their A1C by about 1 to 1.5 percentage points and lost 4.5% to 7.6% of their body weight, compared to minimal changes with a dummy pill.
The New England journal of medicine · NCT05971940
Researchers compared aficamten, a newer heart medication, to metoprolol, a common beta-blocker, in 175 adults with obstructive hypertrophic cardiomyopathy. After 24 weeks, people taking aficamten improved their exercise capacity and symptoms more than those on metoprolol.
The New England journal of medicine · NCT05767346
Surgeons tested a fiber-based light tool that makes parathyroid glands glow during thyroid removal surgery. The technology helped doctors spot more parathyroid glands (3.3 vs. 2.8 on average), but didn't reduce complications like low parathyroid hormone levels after surgery.
JAMA surgery · NCT05022667
Scientists gave adults with overweight or obesity weekly shots of tirzepatide or a placebo for six weeks. People on tirzepatide ate about 500 fewer calories at lunch and reported less hunger and fewer food cravings.
Nature medicine · NCT04311411
LENNY tested the MiniMed 780G automated insulin pump system in 98 children ages 2-6 with type 1 diabetes. Auto mode kept blood sugar in range about 10 percentage points more of the time than manual mode.
The Lancet Diabetes & Endocrinology · NCT05574062
This trial tested bevacizumab plus erlotinib in 83 people with advanced papillary kidney cancer, including the inherited HLRCC form that has no proven treatment. Tumors shrank in 72% of those with HLRCC and 35% of those with sporadic disease.
New England Journal of Medicine · NCT01130519
Researchers tested whether having trained nurses conduct end-of-life care conversations with older adults during emergency department visits would help more patients discuss their wishes with doctors. While patients didn't report more planning discussions overall, doctors documented these important conversations in medical records twice as often when nurses intervened.
JAMA network open · NCT05209880
Significant increase in hemoglobin levels and reduction in markers of red blood cell destruction across a broad population of sickle cell patients, regardless of genotype or prior treatment history.
Blood · NCT04195633
Researchers tested upadacitinib, a pill that blocks inflammation signals, in people with giant cell arteritis. Those taking 15 mg daily were nearly twice as likely to stay in remission compared to those on steroids alone, while needing less steroid treatment overall.
The New England journal of medicine · NCT03725202
Researchers tested an automated insulin delivery system that adjusts insulin doses automatically based on blood sugar readings. After 13 weeks, people using the automated system had better blood sugar control than those using standard insulin injections or pumps.
The New England journal of medicine · NCT05785832
This first-in-human study implanted spinal cord stimulators in 3 adults with type 3 spinal muscular atrophy. After 4 weeks of daily 2-hour stimulation sessions, leg strength rose up to 180%, gait quality improved, and walking endurance increased.
Nature Medicine · NCT05430113
This 138-person study evaluated six low-cost tests for sickle cell disease and beta-thalassemia in clinics in Nepal and Canada. Four of the tests detected severe sickle cell disease accurately; only two detected the beta-thalassemia trait reliably.
The Lancet Regional Health - Southeast Asia · NCT05506358
Two large studies tested depemokimab, a twice-yearly injection, in 528 adults with chronic sinus inflammation and nasal polyps who hadn't improved with standard care. After one year, people taking depemokimab had smaller polyps and less nose blockage than those on placebo.
Lancet (London, England) · NCT05274750
Researchers tested a new cystic fibrosis drug combination (vanzacaftor-tezacaftor-deutivacaftor) against the current standard treatment in 971 people age 12 and older. The new treatment worked just as well at maintaining lung function and only needs to be taken once a day instead of twice.
The Lancet. Respiratory medicine · NCT05033080
Scientists tested whether adding brexpiprazole to sertraline worked better than sertraline alone for adults with PTSD. After 10 weeks, people taking both medicines together had greater improvement in PTSD symptoms than those taking sertraline with a dummy pill.
JAMA psychiatry · NCT04124614
SYMPATICO tested whether combining two oral targeted drugs — ibrutinib and venetoclax — works better than ibrutinib alone for mantle cell lymphoma that came back after first-line treatment. Across 366 adults, the combination kept the cancer at bay for nearly 10 extra months and produced more complete responses.
The Lancet Oncology · NCT03112174
Researchers tested deucravacitinib, a daily pill, in over 1,500 people with moderate to severe plaque psoriasis for up to three years. The treatment kept working without increasing side effects over time, and most common issues were mild infections like colds.
JAMA dermatology · NCT03611751
MT-12 tested a daily under-the-tongue dust-mite immunotherapy tablet in 1,460 children ages 5-11 with dust-mite allergic rhinitis, with or without asthma. After about a year, symptoms and medication use both fell.
The Lancet Regional Health - Europe · NCT04145219
Researchers tested depemokimab, a twice-yearly injection, in people with severe asthma and high levels of eosinophils (a type of white blood cell). The treatment cut asthma flare-ups roughly in half compared to standard care alone.
The New England journal of medicine · NCT04718103
Researchers tested ponsegromab, an antibody that blocks a protein called GDF-15, in people losing weight from cancer. At the highest dose, patients gained about 6 pounds more than those on placebo after 12 weeks, with better appetite and activity levels.
The New England journal of medicine · NCT05546476
Researchers tested mirikizumab, an antibody that blocks a protein called IL-23, in people with moderate-to-severe Crohn's disease who hadn't responded well to other treatments. After one year, 38% of patients on mirikizumab showed healing in their intestines and symptom improvement, compared to 9% on placebo.
Lancet (London, England) · NCT03926130
Piranga tested xalnesiran, a small interfering RNA that silences hepatitis B viral genes, alone or with an immune booster in 281 adults with chronic hepatitis B already suppressed on standard pills. The combination with pegylated interferon cleared the surface antigen in roughly 1 in 4.
New England Journal of Medicine · NCT04225715
This 20-patient trial tested whether elexacaftor-tezacaftor-ivacaftor (Trikafta) helps people with the rare N1303K cystic fibrosis mutation, who have not been approved for the drug. Lung function and weight improved, but the sweat chloride test did not.
The Lancet Respiratory Medicine · NCT03506061
This 37-patient trial tested combined radiation and chemotherapy as an alternative to bladder removal in people with high-grade T1 bladder cancer that failed BCG. At 3 years, 88% kept their bladders.
Journal of Clinical Oncology · NCT00981656
This 50-patient early trial tested mezagitamab — an under-the-skin anti-CD38 antibody — in heavily pretreated multiple myeloma. At the recommended dose, 47% had a tumor response with a median response duration of 22 months.
Blood Neoplasia · NCT03439280
Researchers tested a shorter course of proton beam radiation, guided by specialized imaging, in 39 adults aged 65 and older with glioblastoma. Over half the participants were alive at one year, compared to typical survival of 6-9 months with standard treatment.
The Lancet. Oncology · NCT03778294
Researchers tested two immunotherapy drugs together in people with hard-to-treat thyroid cancers. The combination showed some promise in rare aggressive types but didn't work well enough in the main group studied.
JAMA oncology · NCT03246958
HGB-212 tested betibeglogene autotemcel (beti-cel) gene therapy in 18 people with the most severe beta-thalassemia genotypes. After a one-time infusion, 89% reached transfusion independence with near-normal hemoglobin levels.
The Lancet · NCT03207009
This study tested whether specially designed videos could encourage more Black parents to sign their children up for a research registry. The videos didn't increase sign-ups, but offering a gift card instead of a small pen made parents five times more likely to enroll.
JAMA network open · NCT06138145
This 1,232-person trial in Nigeria tested whether an AI-guided digital stethoscope could detect peripartum cardiomyopathy. The AI tool more than doubled detection of left ventricular dysfunction compared with standard care.
Nature Medicine · NCT05438576
ARTEMIS-UC tested tulisokibart, an IV antibody targeting an inflammatory pathway, in 178 people with moderate-to-severe ulcerative colitis. After 12 weeks, far more on tulisokibart reached clinical remission than on the comparison treatment.
New England Journal of Medicine · NCT04996797
Researchers tested Lu AG09222, an antibody targeting a brain protein called PACAP, in adults with migraine who hadn't responded to at least two other preventive treatments. A single infusion reduced migraine days by about 2 more days per month compared to placebo over the first four weeks.
The New England journal of medicine · NCT05133323
Researchers tested trastuzumab deruxtecan in 122 people with advanced colorectal cancer that had a specific protein called HER2 and had already tried other treatments. In the group receiving the lower dose (5.4 mg/kg), 38% of patients saw their tumors shrink.
The Lancet. Oncology · NCT04744831
Researchers tested delgocitinib cream, a new topical treatment, in adults with moderate to severe chronic hand eczema. After 16 weeks of twice-daily use, about 20-29% of patients achieved clear or almost clear skin, compared to only 7-10% using a plain cream. Side effects were mild and similar between groups.
Lancet (London, England) · NCT04872101
This study followed 124 people with chronic hepatitis B and kidney or liver impairment after switching to tenofovir alafenamide. After 96 weeks, viral suppression held strong with stable kidney function and bone density.
The Lancet Gastroenterology & Hepatology · NCT03180619
LEX-209 compared two 4-factor prothrombin complex concentrate products (Octaplex and Kcentra) for reversing warfarin in 208 patients needing urgent surgery. Both stopped bleeding effectively in about 94% of patients.
JAMA Network Open · NCT02740335
Scientists tested whether combining two drugs—bulevirtide and peginterferon alfa-2a—works better than either drug alone for chronic hepatitis D. Nearly half of patients who got the high-dose combination had no detectable virus six months after finishing treatment, compared to just 12% on bulevirtide alone.
The New England journal of medicine · NCT03852433
Researchers tested maralixibat in children with progressive familial intrahepatic cholestasis (PFIC), a rare genetic liver disease causing severe itching and liver damage. Children taking maralixibat had significantly less itching and lower bile acid levels compared to those on placebo.
The lancet. Gastroenterology & hepatology · NCT03905330
Doctors tested whether ultrasound of the temple arteries could diagnose giant cell arteritis without needing a biopsy. In patients with suspected arteritis, ultrasound correctly identified 54% of confirmed cases. This test could help avoid biopsies in many patients.
Annals of internal medicine · NCT02703922
Researchers tested gantenerumab in people with a rare inherited form of early-onset Alzheimer's disease. The drug reduced several brain proteins linked to damage and inflammation, suggesting it may slow disease processes in this genetic form of Alzheimer's.
JAMA neurology · NCT04623242
SEQUOIA-HCM tested aficamten, a daily oral cardiac myosin inhibitor, in 282 adults with symptomatic obstructive hypertrophic cardiomyopathy. After 24 weeks, exercise capacity improved meaningfully and most secondary measures favored aficamten.
New England Journal of Medicine · NCT05186818
Veterans with PTSD received a mild electrical brain stimulation while experiencing virtual reality scenarios based on war zones. Those who got the real stimulation showed bigger improvements in PTSD symptoms one month later compared to those who got a fake version.
JAMA psychiatry · NCT03372460
This 602-patient trial compared on-site telemedicine hepatitis C care at opioid treatment programs with off-site referral to a specialist. Telemedicine more than doubled cure rates by removing referral barriers.
JAMA · NCT02933970
EPIDYS tested givinostat, a twice-daily oral pill, in 179 ambulatory boys with Duchenne muscular dystrophy already on steroids. After 72 weeks, the decline in stair-climbing time was significantly smaller on givinostat.
The Lancet Neurology · NCT02851797
Researchers analyzed data from over 4,000 kidney disease patients across 21 countries. They found that people exposed to more hot days experienced faster decline in kidney function over time.
The Lancet. Planetary health · NCT03036150
PULSAR tested whether a higher dose of aflibercept (8 mg vs. the standard 2 mg) lets patients with wet age-related macular degeneration stretch their eye injections farther apart. Across 1,011 adults, vision gains were equal — but ~80% of high-dose patients only needed an injection every 12 weeks or longer.
The Lancet · NCT04423718
Researchers compared two immunosuppressant medications, azathioprine and mycophenolate mofetil, in 78 people with myasthenia gravis. Both drugs improved quality of life for more than half of patients. No clear winner emerged, though azathioprine caused more liver problems while mycophenolate caused more stomach issues.
The Lancet. Neurology · NCT03490539
RESPONSE tested seladelpar, a daily pill, in 193 people with primary biliary cholangitis whose liver enzymes had not improved enough on ursodeoxycholic acid. After 12 months, far more on seladelpar reached the biochemical response goal — and itching improved too.
New England Journal of Medicine · NCT04620733
Scientists tested a new drug called BI 690517 in people with chronic kidney disease who were already taking standard medications. The drug reduced protein in urine by up to 39%, a sign that kidney damage may be slowing down.
Lancet (London, England) · NCT05182840
A mobile app helped doctors address the needs of family members whose loved ones were on life support in the ICU. Overall, families using the app reported fewer unmet needs after three days. However, the benefit was mainly seen in White families, not Black families.
JAMA network open · NCT03506438
Researchers tested a structured conversation between dialysis patients, their chosen decision-makers, and a nurse or social worker. Patients and families who had this conversation were better prepared for end-of-life decisions than those who didn't.
JAMA network open · NCT03138564
EXCURSION followed 365 children ages 6-11 with moderate-to-severe asthma on dupilumab — a biweekly under-the-skin injection — for an additional year after the parent study. The drug stayed safe and effective, with no new safety concerns.
The Lancet Respiratory Medicine · NCT03560466
This 440-patient trial added pembrolizumab — an immunotherapy infusion — to standard platinum-pemetrexed chemotherapy for untreated advanced pleural mesothelioma. Adding pembrolizumab modestly extended survival.
The Lancet · NCT02784171
SELECT tested semaglutide, a once-weekly injection, in 17,604 adults with overweight or obesity and existing heart disease but no diabetes. After more than three years on average, semaglutide cut major cardiovascular events compared with placebo, separate from any diabetes benefit.
New England Journal of Medicine · NCT03574597
Researchers tested a mobile app called InCharge Health with 293 people with sickle cell disease. After 24 weeks, patients took their hydroxyurea medication 40% more consistently and had fewer hospital admissions for pain.
Blood advances · NCT04080167
RISE tested TV-46000, a long-acting risperidone injection given monthly or every two months, against an inactive injection in 544 adults with schizophrenia. Both dosing schedules dramatically delayed relapse compared with the inactive injection.
The Lancet Psychiatry · NCT03503318
This 30-patient trial gave tocilizumab — a weekly antibody injection — together with just 8 weeks of steroids for new or relapsed giant cell arteritis. Three-quarters were in steroid-free remission at one year, suggesting much shorter steroid courses may work.
The Lancet Rheumatology · NCT03726749
Scientists tested brexpiprazole in people with Alzheimer's disease who were experiencing agitation. After 12 weeks, those taking the medication showed more improvement in agitation symptoms than those on a sugar pill. The treatment was generally well-tolerated.
JAMA neurology · NCT03548584
Researchers tested avasopasem, a drug designed to protect healthy tissue, combined with high-dose radiation in people with pancreatic cancer that had not spread. The combination showed promising results, with 89-100% of patients responding well at the tested doses.
The Lancet. Oncology · NCT03340974
Researchers tested a new once-weekly insulin called icodec against a daily insulin in people with type 1 diabetes. Both worked equally well at lowering blood sugar, but the weekly insulin caused nearly twice as many low blood sugar episodes.
Lancet (London, England) · NCT04848480
Researchers tested a new vaccine called ChAdOx1-HBV in people with chronic hepatitis B and healthy volunteers. The vaccine was safe and triggered immune responses against the virus, though responses were stronger in healthy people than in those with chronic infection.
JHEP reports : innovation in hepatology · NCT04297917
Researchers tested patisiran, an injection given every 3 weeks, in 360 people with ATTR amyloidosis affecting the heart. After 12 months, people taking patisiran walked farther and reported better quality of life compared to those on placebo.
The New England journal of medicine · NCT03997383
This 40-patient trial added mosunetuzumab — a bispecific antibody that engages T cells against B-cell tumors — to standard CHOP chemotherapy for previously untreated diffuse large B-cell lymphoma. Complete response rate was 85%, and 2-year progression-free survival was 65%.
Blood · NCT03677141
This 110-child trial in Nigeria tested ready-to-use therapeutic food, with or without hydroxyurea, for older children with sickle cell anemia and severe acute malnutrition. After 12 weeks, 39% improved their BMI z-score above the severe-malnutrition cutoff with no serious safety problems.
Blood Advances · NCT03634488
Researchers compared two common hospital antibiotics, cefepime and piperacillin-tazobactam, in over 2,500 adults with acute infections. Both drugs were equally safe for the kidneys, but cefepime was linked to more neurological problems like delirium and confusion.
JAMA · NCT05094154
NEURO-TTRansform tested eplontersen, a once-monthly under-the-skin injection that silences the transthyretin gene, in 168 adults with hereditary ATTR amyloidosis nerve disease. After about a year, transthyretin levels dropped 82% and nerve function and quality of life improved compared with historical comparison.
JAMA · NCT04136184
Researchers tested a combination treatment called NALIRIFOX against a standard treatment in 770 people with pancreatic cancer that had spread. People who got NALIRIFOX lived about two months longer on average.
Lancet (London, England) · NCT04083235
ALLIANCE compared two HIV pills — bictegravir/emtricitabine/tenofovir alafenamide and dolutegravir/emtricitabine/tenofovir disoproxil — in 243 adults with both HIV and hepatitis B. Both suppressed HIV equally well; the bictegravir combo suppressed hepatitis B faster.
The Lancet HIV · NCT03547908
RISE-PD tested IPX203 — an extended-release carbidopa-levodopa capsule — against immediate-release carbidopa-levodopa in 506 people with Parkinson's and motor fluctuations. IPX203 added about 30 minutes more good "on-time" per day with fewer doses.
JAMA Neurology · NCT03670953
EXPLORER-CN tested mavacamten in 81 Chinese patients with symptomatic obstructive hypertrophic cardiomyopathy. The drug dramatically lowered heart-blockage pressure and improved symptoms, matching the results from earlier global trials.
JAMA Cardiology · NCT05174416
VALOR-HCM tested whether mavacamten could keep severely symptomatic obstructive hypertrophic cardiomyopathy patients out of septal reduction surgery. At 56 weeks, only 9% on long-term mavacamten still met criteria for surgery.
JAMA Cardiology · NCT04349072
Researchers tested MDMA combined with therapy in 104 people with moderate to severe PTSD. Those receiving MDMA with therapy saw their PTSD symptoms drop by nearly 24 points on a standard severity scale, compared to about 15 points in the therapy-only group.
Nature medicine · NCT04077437
Researchers tested whether people with chest pain but very low risk of heart disease could safely skip testing. Among 422 low-risk patients, those who deferred testing had fewer unnecessary procedures and no heart attacks or deaths, compared to 1 heart attack and 1 death in the usual testing group.
JAMA cardiology · NCT03702244
Researchers tested lorundrostat, a drug that blocks aldosterone production, in 200 adults whose blood pressure stayed high despite taking multiple medications. After 8 weeks, the 50 mg daily dose lowered systolic blood pressure by about 10 points more than placebo.
JAMA · NCT05001945
GATHER2 tested avacincaptad pegol, a monthly eye injection that blocks part of the immune system, in 448 adults with geographic atrophy — the dry form of late-stage macular degeneration. Over 12 months the drug slowed the growth of vision-loss patches by ~14% compared with sham injections.
The Lancet · NCT04435366
Researchers tested semaglutide (2.4 mg weekly) in 529 people with heart failure and obesity. After one year, those taking semaglutide had fewer heart failure symptoms, could walk farther, and lost more weight than those on inactive treatment.
The New England journal of medicine · NCT04788511
A daily targeted pill reduced the risk of cancer recurrence by 80% compared to placebo in early-stage EGFR-mutated lung cancer patients after surgery — one of the most significant results in thoracic oncology in recent years.
New England Journal of Medicine · NCT02453906
This study tested whether people with rheumatoid arthritis could safely switch back and forth multiple times between the original Humira and a biosimilar version called adalimumab-afzb. After three switches over 22 weeks, blood levels of the drug and side effects were the same in people who switched compared to those who stayed on original Humira the whole time.
The Lancet. Rheumatology · NCT04230213
Researchers tested JNJ-3989, a medication that lowers hepatitis B virus levels, in combination with standard drugs. About 1 in 5 patients taking the higher doses reached targets that could allow them to stop daily pills.
The lancet. Gastroenterology & hepatology · NCT03982186
This study tested whether adding ipilimumab to nivolumab could help patients whose melanoma had grown despite initial immunotherapy. The combination slowed cancer growth better than ipilimumab alone, with 28% of patients seeing their tumors shrink compared to 9% with single-drug treatment.
Nature medicine · NCT03033576
Scientists tested whether insulin pumps could use data from smartwatches to adjust insulin automatically during exercise. Both systems kept blood sugar in a healthy range over 70% of the time and reduced low blood sugar episodes compared to standard treatment.
The Lancet. Digital health · NCT04771403
BRUIN tested pirtobrutinib, a different kind of BTK inhibitor pill, in mantle cell lymphoma patients whose cancer had already come back after the standard BTK drugs (ibrutinib, acalabrutinib, or zanubrutinib). Across 166 patients, more than half saw their tumors shrink, and responses lasted nearly two years on average.
Journal of Clinical Oncology · NCT03740529
Researchers tested tirzepatide, a once-weekly injection, in 938 adults with type 2 diabetes and obesity. After 72 weeks, people taking tirzepatide lost significantly more weight than those on placebo, with most losing at least 5% of their body weight.
Lancet (London, England) · NCT04657003
This Phase 2 study tested retatrutide, a once-weekly injection that activates three gut hormones at once (GLP-1, GIP, and glucagon), in 338 adults with obesity but without diabetes. At 48 weeks, people on the highest dose lost an average of ~24% of their body weight — bigger than what either semaglutide or tirzepatide trials have shown.
New England Journal of Medicine · NCT04881760
CheckMate 401 was a 533-patient real-world trial of nivolumab plus ipilimumab in advanced melanoma, including groups with poorer outcomes (brain metastases, ocular/uveal melanoma, mucosal melanoma). The 24-month survival rate was 63% overall and 71% in those with brain metastases.
Journal of Clinical Oncology · NCT02599402
TRAILBLAZER-ALZ 2 tested donanemab, a monthly IV antibody that targets amyloid plaques in the brain, in 1,736 people with early symptomatic Alzheimer's. After 76 weeks, donanemab slowed cognitive decline most in people whose disease was caught earliest.
JAMA · NCT04437511
STOP-CA tested whether daily atorvastatin protects the heart in 300 lymphoma patients receiving anthracycline chemotherapy. After a year, far fewer patients on atorvastatin had a meaningful drop in heart pumping function.
JAMA · NCT02943590
ACeD was a first-in-human safety trial of KAN-101, an IV treatment designed to retrain the immune system to tolerate gluten, in 41 adults with celiac disease. The drug was safe at all doses tested, with no serious side effects.
The Lancet Gastroenterology & Hepatology · NCT04248855
Pregnant people at risk of early delivery watched short animated videos on their phones about preterm birth. By 25 weeks, those who watched the videos scored 15 percentage points higher on knowledge tests and felt much more prepared to make decisions about their baby's care compared to those who received standard written materials.
JAMA pediatrics · NCT04093492
Researchers tested dupilumab in people with COPD who had high levels of certain immune cells called eosinophils. Those who received dupilumab had 30% fewer flare-ups, better breathing, and improved quality of life compared to those on standard treatment alone.
The New England journal of medicine · NCT03930732
Researchers tested bulevirtide, an injection given daily, in people with chronic hepatitis D (a liver virus that makes hepatitis B worse). After 48 weeks, nearly half of patients who got the treatment saw their virus levels drop significantly and their liver inflammation markers return to normal.
The New England journal of medicine · NCT03852719
LUCENT-1 and LUCENT-2 tested mirikizumab, an antibody that blocks IL-23, as both a starter and maintenance treatment for moderately to severely active ulcerative colitis. It became the first IL-23 inhibitor approved for ulcerative colitis.
New England Journal of Medicine · NCT03518086
Researchers compared ketamine infusions to electroconvulsive therapy in 403 people with depression that hadn't responded to other treatments. More patients improved with ketamine (55%) than with ECT (41%), and ketamine didn't cause the memory problems seen with ECT.
The New England journal of medicine · NCT03113968
This trial tested rimegepant — a fast-dissolving tablet that blocks the CGRP migraine pathway — for treating a single migraine attack in 1,431 adults in China and South Korea. Twice as many people on rimegepant were pain-free at 2 hours.
The Lancet Neurology · NCT04574362
TitAIN tested secukinumab, a monthly injection that blocks the IL-17 inflammation pathway, in 52 people with giant cell arteritis. Far more on secukinumab were in sustained remission at 28 weeks while tapering off steroids.
The Lancet Rheumatology · NCT03765788
Researchers tested valbenazine in 128 adults with Huntington's disease who had involuntary movements called chorea. After 12 weeks, people taking valbenazine had significantly less chorea than those taking a placebo. The treatment was generally well tolerated.
The Lancet. Neurology · NCT04102579
Researchers tested a new once-daily pill combining three drugs (vanzacaftor, tezacaftor, and deutivacaftor) in adults with cystic fibrosis. People taking the combination saw significant improvements in lung function and breathing ability compared to those on existing treatments or placebo.
The Lancet. Respiratory medicine · NCT03912233
U-EXCEL and the companion U-EXCEED trial tested upadacitinib, a once-daily oral pill, as a starter treatment in 1,021 adults with moderately to severely active Crohn's. The pill brought significantly more patients into remission and healed visible intestinal inflammation within 12 weeks.
New England Journal of Medicine · NCT03345849
RAISE tested zilucoplan, a daily self-injection that blocks complement — an immune pathway that damages nerve-muscle connections in myasthenia gravis — in 174 adults whose disease was driven by AChR antibodies.
The Lancet Neurology · NCT04115293
MOUNTAINEER tested tucatinib plus trastuzumab — a daily targeted pill plus an IV antibody — in 117 people with HER2-positive metastatic colorectal cancer that had failed chemotherapy. Tumors shrank in 38%.
The Lancet Oncology · NCT03043313
MycarinG tested rozanolixizumab, a weekly under-the-skin antibody infusion that lowers harmful antibodies, in 200 adults with generalized myasthenia gravis. Both doses produced a meaningful reduction in daily-life symptoms.
The Lancet Neurology · NCT03971422
This pooled analysis of three trials looked at 552 adolescents ages 12-17 with moderate-to-severe atopic dermatitis on upadacitinib — a daily oral JAK inhibitor pill. About 7-8 in 10 had at least 75% improvement in eczema at 16 weeks on the higher dose.
JAMA Dermatology · NCT03607422
Researchers tested rozanolixizumab, a weekly injection that blocks a protein called the neonatal Fc receptor. After 6 weeks, patients with myasthenia gravis had significantly fewer daily symptoms compared to those who received a placebo. The most common side effects were headache and diarrhea.
The Lancet. Neurology · NCT04124965
Researchers tested rozanolixizumab, a weekly injection that blocks a protein called the neonatal Fc receptor, in 200 people with myasthenia gravis. After six weeks, those receiving the drug showed significantly greater improvement in daily activities compared to those on placebo.
The Lancet. Neurology · NCT04650854
Researchers tested mirvetuximab soravtansine in 106 women with ovarian cancer that stopped responding to platinum chemotherapy and had high levels of a protein called folate receptor alpha. About one in three patients saw their tumors shrink, and responses lasted a median of nearly 7 months.
Journal of clinical oncology : official journal of the American Society of Clinical Oncology · NCT04296890
Researchers tested tezepelumab, an antibody injection given every 4 weeks, in people with severe asthma that wasn't well controlled. Over 2 years, people taking tezepelumab had 58% fewer asthma attacks compared to those on placebo, and the treatment was well tolerated.
The Lancet. Respiratory medicine · NCT03706079
ELEVATE UC 52 and ELEVATE UC 12 tested etrasimod, a once-daily pill that limits inflammatory immune cells from entering the gut, in 1,141 adults with moderately to severely active ulcerative colitis. It was the first oral pill of its class approved for ulcerative colitis.
The Lancet · NCT03996369
Scientists tested an experimental drug called RO7020531 in healthy volunteers and people with chronic hepatitis B. The drug was designed to boost the immune system's response to the virus. All tested doses were safe and well-tolerated, with mostly mild and temporary side effects.
The Lancet. Infectious diseases · NCT02956850
Scientists compared two types of walking exercise in stroke survivors with walking problems. Those who did short bursts of fast walking (high-intensity interval training) walked 71 meters farther after 12 weeks, while those doing steady moderate walking walked 27 meters farther.
JAMA neurology · NCT03760016
Researchers tested whether a biosimilar version of natalizumab (called PB006) works as well as the original drug (Tysabri) for relapsing-remitting multiple sclerosis. After 24 weeks, patients on both versions had nearly identical numbers of new brain lesions and similar side effects.
JAMA neurology · NCT04115488
Researchers tested whether insulin degludec works as well as insulin detemir for pregnant women with type 1 diabetes. Both insulins kept blood sugar levels similar before delivery, and degludec was just as safe.
The lancet. Diabetes & endocrinology · NCT03377699
Researchers tested efanesoctocog alfa, a new once-weekly injection for people with severe hemophilia A. Patients who switched from their previous prevention treatment had 77% fewer bleeding episodes—dropping from about 3 bleeds per year to less than 1.
The New England journal of medicine · NCT04161495
Scientists studied 163 people with sickle cell disease who received stem cell transplants from donors. They tracked how often patients had pain crises before and after the procedure. The transplants dramatically reduced these painful episodes.
Blood advances · NCT03263559
This 274-patient trial tested rocatinlimab, an antibody injection that targets the OX40 immune pathway, in adults with moderate-to-severe atopic dermatitis (eczema). All four dose schedules cut eczema severity scores by about half by week 16.
The Lancet · NCT03703102
CLARITY-AD tested lecanemab, an antibody given as an IV infusion every two weeks that clears amyloid plaques from the brain, in 1,795 people with early Alzheimer's disease. After 18 months, lecanemab slowed the loss of memory and daily function compared with placebo.
New England Journal of Medicine · NCT03887455
Researchers tested a pill called ENT-01 in 150 people with Parkinson's disease who had constipation. People taking ENT-01 had about 3 bowel movements per week compared to 1 per week with a fake pill. The treatment also helped with stool consistency and reduced laxative use.
Annals of internal medicine · NCT03781791
Researchers tested whether trientine works as well as penicillamine (the standard treatment) for maintaining copper balance in adults with stable Wilson disease. After 24 weeks, trientine controlled copper levels just as well as penicillamine and caused fewer serious side effects.
The lancet. Gastroenterology & hepatology · NCT03539952
This trial compared two patient-centered care models — modified directly observed therapy and patient navigation — for hepatitis C treatment in 755 active people who inject drugs. Both approaches achieved high cure rates with sofosbuvir-velpatasvir.
The Lancet Gastroenterology & Hepatology · NCT02824640
B-Clear tested bepirovirsen, a weekly antisense injection that lowers hepatitis B viral proteins, in 457 adults with chronic hepatitis B. About one in ten reached a sustained loss of the surface antigen — a major step toward functional cure.
New England Journal of Medicine · NCT04449029
This 17-patient dose-escalation study tested mitapivat — a daily oral pill that activates a red-cell enzyme — in adults with sickle cell disease. Hemoglobin rose by an average of 1.2 g/dL on the 50 mg dose, with about 56% having a clinically meaningful response.
Blood · NCT04000165
COMP360 tested whether a single 25-mg dose of psilocybin — the active compound in psychedelic mushrooms — given alongside structured psychological support, helps adults with treatment-resistant depression. Across 233 patients at 22 sites, the 25-mg dose lifted depression scores significantly more than a tiny 1-mg control at 3 weeks.
New England Journal of Medicine · NCT03775200
Adding Keytruda (pembrolizumab) to chemotherapy reduced the risk of death by 27% in patients with triple-negative breast cancer — one of the hardest subtypes to treat and a population with historically limited options.
New England Journal of Medicine · NCT03036488
Researchers tested a pill called obefazimod (ABX464) in people with moderate to severe ulcerative colitis who hadn't responded to other treatments. After 8 weeks, all three dose levels improved symptoms significantly more than placebo, with patients seeing their disease activity scores drop by about 3 points compared to about 2 points with placebo.
The lancet. Gastroenterology & hepatology · NCT04023396
Researchers tested rituximab in people newly diagnosed with myasthenia gravis, a condition where muscles get weak. After 16 weeks, 71% of people who got rituximab had minimal symptoms on low steroid doses, compared to 29% with placebo.
JAMA neurology · NCT02950155
Researchers tested luspatercept in adults with beta-thalassemia who don't need regular blood transfusions but have low hemoglobin levels. 77% of patients taking luspatercept had meaningful increases in hemoglobin levels, compared to none in the placebo group.
The Lancet. Haematology · NCT03342404
Scientists tested a pill combining two hepatitis C medicines in 37 Japanese adults who had liver scarring. Every patient cleared the virus after 12 weeks of treatment.
Hepatology research : the official journal of the Japan Society of Hepatology · NCT04112303
The ADVOR trial tested whether adding acetazolamide (a diuretic pill) to standard diuretic treatment helps people hospitalized with heart failure remove excess fluid faster. Patients who received acetazolamide were significantly more likely to successfully remove fluid within three days compared to those receiving standard treatment alone.
The New England journal of medicine · NCT03505788
Researchers tested tofersen, a drug designed to lower levels of a harmful protein in people with ALS caused by SOD1 gene mutations. The drug successfully reduced the protein in spinal fluid, but didn't improve muscle function or breathing over 28 weeks. People who started the drug earlier showed hints of better outcomes at one year.
The New England journal of medicine · NCT03070119
Two large studies tested roflumilast cream applied once daily for 8 weeks in people with plaque psoriasis. About 40% of patients using the cream had clear or almost clear skin, compared to only 6-7% using a plain cream. The treatment also reduced itching significantly.
JAMA · NCT04211389
Researchers tested a single pill combining aspirin, ramipril, and atorvastatin in people who had a heart attack in the past 6 months. The polypill cut the risk of death, another heart attack, stroke, or urgent heart procedures by 24% compared to usual care.
The New England journal of medicine · NCT02596126
Researchers compared dimethyl fumarate (Tecfidera) with interferon beta-1a in children and teens with relapsing-remitting multiple sclerosis. After nearly two years, kids taking dimethyl fumarate had about half as many relapses and fewer new brain lesions on MRI scans.
JAMA network open · NCT02283853
Researchers tested a three-drug combination in 65 people with melanoma that had spread to the brain and carried a specific gene change (BRAF mutation). The treatment shrank brain tumors in 42% of patients.
The Lancet. Oncology · NCT03625141
CREDO2 tested olokizumab, an interleukin-6 blocker, against both an inactive comparison and adalimumab (Humira) in 1,648 adults with rheumatoid arthritis still active on methotrexate. At 12 weeks, olokizumab matched adalimumab for joint improvement.
New England Journal of Medicine · NCT02760407
Researchers compared ublituximab (a drug given through an IV) to teriflunomide (a pill) in people with relapsing multiple sclerosis. Those who got ublituximab had 59% fewer relapses and far fewer new brain lesions over nearly two years.
The New England journal of medicine · NCT03277261
SURMOUNT-1 tested tirzepatide, a weekly injection that activates two gut hormones (GLP-1 and GIP) instead of just one, in 2,539 adults with overweight or obesity but without diabetes. Over 72 weeks, the highest dose produced an average ~22.5% body weight loss — the largest effect ever shown in a weight-loss medication trial.
New England Journal of Medicine · NCT04184622
KRYSTAL-1 tested adagrasib, a twice-daily oral pill targeting the KRAS G12C mutation, in 116 people with previously treated advanced non-small cell lung cancer. The drug shrank tumors in nearly half of patients and had a meaningful effect on cancer that had spread to the brain.
New England Journal of Medicine · NCT03785249
DESTINY-Breast04 tested trastuzumab deruxtecan in a population doctors used to consider untreatable with HER2-targeted therapy: women whose tumors had only low levels of HER2. Across 557 women, the drug roughly doubled the time before cancer progressed and improved overall survival.
New England Journal of Medicine · NCT03734029
Researchers tested a targeted ultrasound treatment that uses MRI to guide heat energy to destroy cancer in one area of the prostate. In men with intermediate-risk prostate cancer, 88% had no cancer in the treated area after two years.
The Lancet. Oncology · NCT01657942
Researchers tested onasemnogene abeparvovec, a one-time gene therapy, in babies at risk for spinal muscular atrophy type 2. When given before symptoms appeared, all 15 babies stood on their own, 14 walked independently, and none needed breathing machines or feeding tubes.
Nature medicine · NCT03505099
SPIRIT 1 and SPIRIT 2 tested a once-daily relugolix pill combined with low-dose hormones in 1,261 women with moderate-to-severe pain from endometriosis. After 24 weeks, more women on the combination pill reported significant pain relief during periods and on other days.
The Lancet · NCT03204318
ANCHOR enrolled 4,446 people living with HIV who had biopsy-proven anal high-grade pre-cancer lesions. After about two years, treating those lesions cut the rate of progression to anal cancer compared with watchful waiting.
New England Journal of Medicine · NCT02135419
Researchers compared two biologic drugs, ustekinumab and adalimumab, in people with moderate to severe Crohn's disease who hadn't taken biologics before. After one year, both drugs worked about equally well at controlling symptoms.
Lancet (London, England) · NCT03464136
SHINE tested whether adding ibrutinib (a daily targeted pill) to standard chemoimmunotherapy as the very first treatment for mantle cell lymphoma keeps the cancer at bay longer. In 523 patients aged 65 and older, the combination delayed disease progression by about 2.3 years on average — but did not extend overall survival, and side effects were heavier.
New England Journal of Medicine · NCT01776840
Researchers tested upadacitinib, a pill taken once daily, in people with moderate to severe ulcerative colitis. In the induction studies, about 26-34% of patients taking upadacitinib achieved remission after 8 weeks, compared to 4-5% on placebo. In the maintenance study, 42-52% of patients on upadacitinib stayed in remission at one year versus 12% on placebo.
Lancet (London, England) · NCT03653026
This study tested tazemetostat, a daily oral pill, in 74 people with mesothelioma whose tumors had lost the BAP1 gene and had progressed after chemotherapy. About half had stable disease or shrinkage at 12 weeks.
The Lancet Oncology · NCT02860286
ADVANCE and the companion MOTIVATE trial tested risankizumab, an IV antibody that blocks IL-23, as a starter treatment for moderately to severely active Crohn's disease. Across the program, risankizumab brought significantly more people into remission than placebo within 12 weeks.
The Lancet · NCT03104413
Researchers tested risankizumab, a drug that blocks a protein called IL-23, in people with moderate to severe Crohn's disease who hadn't responded well to other treatments. After 12 weeks, about 40-45% of patients achieved remission compared to 20-25% on placebo.
Lancet (London, England) · NCT03105128
BRAVE-AA1 and BRAVE-AA2 tested baricitinib, a daily pill, in 1,200 adults with severe alopecia areata who had lost at least half their scalp hair. After 36 weeks, more than a third of people on the higher dose had near-full scalp regrowth.
New England Journal of Medicine · NCT03899259
BRAVE-AA1 and BRAVE-AA2 tested baricitinib, a daily oral JAK inhibitor pill, in 1,200 adults with severe scalp hair loss from alopecia areata. After 36 weeks, more than a third of those on the higher dose had near-complete regrowth.
New England Journal of Medicine · NCT03570749
Scientists tested ganaxolone in children with CDKL5 deficiency disorder, a rare genetic condition causing hard-to-control seizures starting in infancy. Kids taking ganaxolone had 31% fewer major seizures compared to 7% fewer in those taking placebo.
The Lancet. Neurology · NCT03572933
Researchers tested whether people with hepatitis C could take sofosbuvir-velpatasvir at home for 12 weeks without regular doctor visits or lab tests. 95% of patients cleared the virus successfully, showing that minimal monitoring works as well as standard care.
The lancet. Gastroenterology & hepatology · NCT03512210
Researchers compared rucaparib (a PARP inhibitor pill) to standard chemotherapy in 349 women with ovarian cancer that had returned and carried a BRCA1 or BRCA2 mutation. Women taking rucaparib went longer without their cancer growing compared to those on chemotherapy.
The Lancet. Oncology · NCT02855944
DESTINY-Breast03 tested trastuzumab deruxtecan, an antibody linked to chemotherapy that delivers the drug directly to HER2-positive cancer cells, head-to-head against trastuzumab emtansine — the previous standard — in 524 women with HER2-positive metastatic breast cancer.
New England Journal of Medicine · NCT03529110
Researchers tested valoctocogene roxaparvovec, a one-time gene therapy, in 134 men with severe hemophilia A. After one year, patients made their own clotting factor and needed 99% less replacement treatment. Bleeding episodes dropped by 84%.
The New England journal of medicine · NCT03370913
Scientists tested iberdomide, a new pill taken daily alongside standard lupus medications. After 24 weeks, 54% of patients on the highest dose showed meaningful improvement in disease activity, compared to 35% on placebo.
The New England journal of medicine · NCT03161483
Scientists tested CAP-1002, a treatment made from heart-derived cells, in boys and young men with advanced Duchenne muscular dystrophy. Patients who received four infusions over a year maintained upper body strength better than those on placebo, and some heart measures also improved.
Lancet (London, England) · NCT03406780
GALAHAD tested niraparib, a daily oral PARP inhibitor pill, in 289 men with metastatic castration-resistant prostate cancer carrying DNA-repair gene defects. About a third of those with BRCA mutations had tumor shrinkage.
The Lancet Oncology · NCT02854436
This 2,050-adult trial tested whether sharing APOL1 kidney-risk gene results helped Black patients with high blood pressure. Patients who learned they had high-risk genotypes had bigger drops in blood pressure and more kidney screening at 3 and 12 months.
JAMA Network Open · NCT02234063
Scientists tested a film that dissolves under the tongue containing dexmedetomidine in 380 adults experiencing agitation from bipolar disorder. Both doses tested reduced agitation scores significantly more than placebo within 20 minutes, with effects lasting at least 2 hours.
JAMA · NCT04276883
Researchers tested a one-time gene therapy called lovo-cel in 35 people with severe sickle cell disease. All 25 patients who could be evaluated stopped having severe pain crises after treatment, compared to a typical 3 to 14 crises per year before.
The New England journal of medicine · NCT02140554
SURPASS-5 tested whether adding weekly tirzepatide to existing insulin glargine improves blood sugar in 475 adults with type 2 diabetes. After 40 weeks, A1c dropped about 1.5 points more on tirzepatide and weight dropped about 7-10 kg more.
JAMA · NCT04039503
Researchers tested trametinib, a pill that blocks cancer growth signals, against standard treatments in 260 people with low-grade ovarian cancer that had returned. People taking trametinib went nearly twice as long without their cancer worsening—13 months compared to 7 months.
Lancet (London, England) · NCT02101788
HGB-207 tested betibeglogene autotemcel (beti-cel), a one-time gene therapy, in 23 people with transfusion-dependent beta-thalassemia who do not have the most severe genotype. Most stopped needing red-cell transfusions entirely.
New England Journal of Medicine · NCT02906202
TENAYA tested faricimab, an eye injection that blocks two different growth signals at once, in 671 adults with wet age-related macular degeneration. Vision gains matched what people get on the previous standard drug aflibercept — but many faricimab patients only needed an injection every 16 weeks instead of every 8.
The Lancet · NCT03823287
Scientists tested vamorolone, a drug designed to reduce inflammation and muscle damage, in 41 boys with Duchenne muscular dystrophy over 30 months. Boys on vamorolone maintained muscle strength similar to standard steroid treatment but grew normally instead of experiencing growth delays.
JAMA network open · NCT03038399
SUNFISH tested risdiplam, a daily liquid taken by mouth, in 180 people ages 2-25 with type 2 or non-walking type 3 spinal muscular atrophy. After 12 months, motor function improved on risdiplam compared with the inactive liquid.
The Lancet Neurology · NCT02908685
Researchers tested a new way to treat irregular heartbeat called pulsed field ablation. In 38 patients, the treatment successfully isolated all problem areas in the heart without causing major complications like nerve damage, throat injury, or stroke within 30 days.
Circulation. Arrhythmia and electrophysiology · NCT04198701
Effisayil-1 tested spesolimab, a single IV dose that blocks the IL-36 receptor, in 53 adults during an active flare of generalized pustular psoriasis — a rare and dangerous skin condition where painful pus-filled bumps cover the body. Most patients had visible pustules clear within a week.
New England Journal of Medicine · NCT03782792
Researchers tested daprodustat, a pill, against standard injections for treating anemia in people on dialysis. The pill worked just as well at raising hemoglobin levels and had similar heart safety over 2.5 years.
The New England journal of medicine · NCT02879305
Researchers tested whether edoxaban (a newer blood thinner pill) works as well as warfarin in patients with atrial fibrillation who had their aortic valve replaced through a catheter. Edoxaban worked similarly for preventing strokes and clots, but caused more bleeding, especially from the gut.
The New England journal of medicine · NCT02943785
This 30-patient trial combined an experimental peptide vaccine targeting IDO and PD-L1 with nivolumab in untreated metastatic melanoma. Tumors shrank in 80% of patients, with complete responses in 43% — well above usual rates with nivolumab alone.
Nature Medicine · NCT03047928
KIDCARE compared infliximab with a second IVIG infusion in 103 children whose Kawasaki disease did not respond to the first IVIG. Infliximab worked faster, shortened hospital stays, and caused less anemia.
The Lancet Child & Adolescent Health · NCT03065244
Researchers tested a stomach balloon that can be adjusted in size to help people lose weight. After 32 weeks, people with the balloon lost an average of 15% of their body weight, compared to 3% in those who only changed their diet and exercise habits.
Lancet (London, England) · NCT02812160
This early study followed 18 men with hemophilia A who received a one-time IV infusion of an AAV gene therapy designed to make Factor VIII. Most kept producing the missing clotting factor more than two years later, and bleeding episodes dropped sharply.
New England Journal of Medicine · NCT03003533
SURPASS-4 compared weekly tirzepatide injections with daily insulin glargine in 2,002 adults with type 2 diabetes and high cardiovascular risk. Tirzepatide lowered A1c more, caused less low blood sugar, and did not raise heart-event risk.
The Lancet · NCT03730662
UNWIND tested escitalopram or aerobic exercise versus an inactive pill for anxiety in 128 adults with stable coronary heart disease. Escitalopram reduced anxiety symptoms most; exercise had a smaller, less consistent benefit.
JAMA Psychiatry · NCT02516332
Researchers tested tezepelumab, an antibody that blocks a protein called TSLP, in people with moderate-to-severe asthma. After 28 weeks, the drug reduced inflammation-causing eosinophils in the airways by 85% compared to placebo and improved airway responsiveness.
The Lancet. Respiratory medicine · NCT03688074
EMPEROR-Preserved tested empagliflozin, a once-daily diabetes pill, in 5,988 people with heart failure and a normal-pumping heart (preserved ejection fraction). It was the first large trial to show a clear benefit for this group, who had no proven treatments for years.
New England Journal of Medicine · NCT03057951
This 2,838-adult trial compared a 3-antigen hepatitis B vaccine (PreHevbrio) with the older single-antigen vaccine (Engerix-B) in adults 18-45. After two doses, 90% on the new vaccine had protective antibody levels vs. 52% on the old vaccine.
JAMA Network Open · NCT03408730
Researchers tested a one-time gene therapy called onasemnogene abeparvovec in 33 babies with spinal muscular atrophy type 1. Nearly all babies (97%) survived without needing permanent breathing support at 14 months, and 44% could sit independently — outcomes that almost never happen without treatment.
The Lancet. Neurology · NCT03461289
Researchers tested BI 695501, a biosimilar version of Humira (adalimumab), in 147 adults with moderate to severe Crohn's disease. After 4 weeks, 90% of patients taking BI 695501 and 94% taking original Humira saw their symptoms improve by a meaningful amount. Both treatments worked similarly with comparable side effects.
The lancet. Gastroenterology & hepatology · NCT02871635
Researchers tested ozanimod, a pill taken once daily, in adults with moderate to severe ulcerative colitis. After 10 weeks, 18% of patients taking ozanimod were in remission compared to 6% taking a placebo. After one year of continued treatment, 37% were in remission compared to 19% on placebo.
The New England journal of medicine · NCT02435992
Researchers tested tebentafusp, a treatment that redirects immune cells to attack melanoma, in 378 patients with metastatic uveal melanoma (a rare eye cancer). After one year, 73% of patients who received tebentafusp were alive, compared to 59% who received standard treatments.
The New England journal of medicine · NCT03070392
This extension study followed 57 children ages 2-5 with cystic fibrosis homozygous for the F508del mutation on lumacaftor-ivacaftor for up to 96 weeks. The drug was generally safe, and gains in sweat chloride, growth, and pancreatic function held up.
The Lancet Respiratory Medicine · NCT03125395
Researchers tested a clot-busting drug called staphylokinase against the standard treatment alteplase in stroke patients treated within 4.5 hours. Half the patients who got staphylokinase recovered well at 90 days, compared to 40% with alteplase. Serious side effects were less common with staphylokinase.
The Lancet. Neurology · NCT03151993
Scientists tested tolebrutinib, a pill that blocks a protein involved in brain inflammation, in 130 people with relapsing MS. After 12 weeks, the highest dose (60 mg) reduced new brain lesions by 87% compared to placebo, with minimal side effects.
The Lancet. Neurology · NCT03889639
This 271-patient trial tested whether elexacaftor-tezacaftor-ivacaftor (Trikafta) added benefit for people already on older CF modulators. After 8 weeks, lung function and sweat chloride improved on the triple pill compared with the older combo.
New England Journal of Medicine · NCT04058353
Researchers tested atogepant, a daily pill that blocks a protein linked to migraines. People taking 60 mg had about 4 fewer migraine days per month compared to 2.5 fewer days with a sugar pill. Side effects included constipation and nausea.
The New England journal of medicine · NCT03777059
SURPASS-2 compared tirzepatide head-to-head with semaglutide — two of the most-prescribed weekly diabetes injections — in 1,879 adults with type 2 diabetes. After 40 weeks, tirzepatide lowered blood sugar and weight more than semaglutide at every dose tested.
New England Journal of Medicine · NCT03987919
This 117-person study assigned older adults with hypertension to a 52-week aerobic exercise program or education only. APOE4 carriers — people at highest genetic Alzheimer's risk — saw real gains in hippocampus blood flow with exercise; non-carriers did not.
Journal of Cerebral Blood Flow and Metabolism · NCT02000583
Doctors tested a combination of radiation therapy with two immunotherapy drugs—pembrolizumab and trametinib—in people whose pancreatic cancer came back after surgery. Patients who received this combination lived about 2.5 months longer than those who received radiation plus standard chemotherapy.
The Lancet. Oncology · NCT02704156
Researchers tested a combination of brentuximab vedotin with three chemotherapy drugs (ifosfamide, carboplatin, and etoposide) in 45 people whose Hodgkin lymphoma came back or didn't respond to first treatment. After two cycles, 74% of patients had no signs of cancer on scans.
The Lancet. Haematology · NCT02227199
Researchers tested whether pimavanserin could prevent psychosis from returning in people with dementia caused by Alzheimer's, Parkinson's, Lewy body disease, and other conditions. Patients who improved on the drug were less likely to have their symptoms come back compared to those who switched to a sugar pill.
The New England journal of medicine · NCT03325556
Researchers tested rFIXFc, a long-acting clotting factor, in 33 children with severe hemophilia B who had never received treatment before. Only 1 patient (3%) developed an inhibitor, a complication where the immune system blocks the medicine. Most children on preventive treatment had about 1 bleed per year.
Blood advances · NCT02234310
ADAPT tested efgartigimod, an antibody fragment that lowers harmful autoantibodies in the blood, in 167 adults with generalized myasthenia gravis. The drug works by blocking a recycling receptor that keeps disease-driving antibodies in circulation.
The Lancet Neurology · NCT03669588
ARROW tested pralsetinib, a daily oral targeted pill, in people with non-small-cell lung cancer carrying a rare RET gene fusion. Tumors shrank in 6 of 10 previously treated patients and 7 of 10 untreated patients.
The Lancet Oncology · NCT03037385
Tezacaftor-ivacaftor, a twice-daily pill combination, slowed the rate of lung function decline by 62% compared to untreated patients over two years. The treatment was generally safe and well-tolerated in over 1,000 people with cystic fibrosis aged 12 and older.
The Lancet. Respiratory medicine · NCT02565914
Researchers tested efgartigimod, a treatment designed to lower harmful antibodies, in people with myasthenia gravis. After four weekly infusions, 68% of patients saw meaningful improvement in daily activities lasting at least a month, compared to 30% on placebo.
The Lancet. Neurology · NCT03770403
Veterans with obesity or overweight used fitness trackers with game features and weekly cash penalties if they missed step goals. Those with cash penalties walked about 1,200 more steps per day during the 12-week program compared to basic tracker users, but the benefit disappeared once penalties stopped.
JAMA network open · NCT03563027
Scientists tested TAK-101, tiny particles containing gluten protein, in people with celiac disease. During a two-week gluten challenge, those who received TAK-101 showed 88% less immune response to gluten compared to those who got a fake treatment. The treatment also helped protect the intestinal lining from damage.
Gastroenterology · NCT03738475
Doctors tested whether giving bevacizumab 4 days before chemotherapy works better than giving both on the same day in 230 people with metastatic colorectal cancer. People who got bevacizumab first lived about 6 months longer and had fewer severe side effects.
JAMA network open · NCT01718873
CodeBreaK 100 tested sotorasib, a daily oral pill targeting a specific cancer mutation called KRAS G12C, in 126 people with advanced non-small cell lung cancer who had previously received chemotherapy and immunotherapy. Tumors shrank in over a third of patients on a once-daily pill.
New England Journal of Medicine · NCT03600883
Scientists tested whether giving eptinezumab through an IV during a migraine attack could stop the pain faster than a dummy treatment. People who got eptinezumab had their headache pain stop in half the time compared to those who got the dummy treatment.
JAMA · NCT04152083
APOLLO tested whether adding daratumumab to pomalidomide and dexamethasone improved outcomes in 304 people with previously treated multiple myeloma. The triplet nearly doubled the time before disease progressed.
The Lancet Oncology · NCT03180736
Researchers tested whether baricitinib, a pill that calms immune overactivity, could keep working for adults with moderate to severe eczema over 68 weeks. About half of people who responded early stayed clear or nearly clear throughout the study.
JAMA dermatology · NCT03334435
STR1VE tested onasemnogene abeparvovec (Zolgensma), a one-time IV gene therapy, in 22 infants with SMA type 1. At 14 months, 91% were alive without permanent ventilation, and most learned to sit independently — outcomes never seen in untreated infants.
The Lancet Neurology · NCT03306277
Researchers tested lumasiran, an RNA-based treatment, in people with primary hyperoxaluria type 1 (PH1), a rare genetic disease where the liver makes too much oxalate, leading to kidney stones and kidney failure. After 6 months, lumasiran reduced urinary oxalate by 65% compared to placebo, and 84% of patients reached normal or near-normal levels.
The New England journal of medicine · NCT03681184
Scientists tested a harmless bacteria from healthy skin called Staphylococcus hominis A9 on adults with eczema. When applied to the skin for one week, it was safe and reduced harmful bacteria that make eczema worse.
Nature medicine · NCT03151148
JADE COMPARE tested abrocitinib, a once-daily oral pill, against placebo and against dupilumab — the leading injectable for atopic dermatitis — in 838 adults with moderate-to-severe disease. The higher dose of abrocitinib relieved itch faster than dupilumab in the first weeks of treatment.
New England Journal of Medicine · NCT03720470
Researchers tested enfortumab vedotin in 608 people with advanced bladder cancer whose disease had worsened after standard treatments. People who received enfortumab vedotin lived nearly 4 months longer on average than those who received standard chemotherapy.
The New England journal of medicine · NCT03474107
STEP 1 tested semaglutide, a once-weekly injection (the same drug sold as Wegovy for weight management and Ozempic for diabetes), in 1,961 adults with overweight or obesity but without type 2 diabetes. Over 68 weeks, people on semaglutide lost an average of about 15% of their body weight — roughly 34 pounds for someone starting at 230.
New England Journal of Medicine · NCT03548935
Scientists tested an oral medicine called risdiplam in 21 infants with type 1 spinal muscular atrophy, a severe muscle-wasting disease. Seven of 17 infants on the higher dose gained the ability to sit without support for at least 5 seconds—a milestone these babies typically never reach.
The New England journal of medicine · NCT02913482
Scientists tested a drug combination called KarXT (xanomeline-trospium) in 182 people with schizophrenia. After 5 weeks, people taking KarXT showed significantly greater improvement in symptoms compared to those taking a sugar pill.
The New England journal of medicine · NCT03697252
Researchers tested four different drugs for people with papillary kidney cancer that had spread. Cabozantinib kept the cancer from growing for a median of 9 months compared to 5.6 months with sunitinib, the standard treatment at the time.
Lancet (London, England) · NCT02761057
ADVOCATE tested avacopan, a daily pill that blocks part of the inflammatory cascade, in 331 people with ANCA-associated vasculitis. The goal was to reduce the long-term steroid doses that cause weight gain, infections, and bone loss.
New England Journal of Medicine · NCT02994927
Scientists tested omidubicel, a lab-grown cord blood product, in 16 young people with severe sickle cell disease. The treatment helped cells take hold quickly—in just 7 days on average—and 85% of patients in the main group were alive and disease-free after 4 years.
Blood advances · NCT01590628
Scientists tested two different amounts of gluten in 14 adults with celiac disease to see which blood tests and other measures could detect immune reactions. They found that a blood marker called interleukin-2 detected gluten exposure faster and more sensitively than traditional biopsies.
Gastroenterology · NCT03409796
Researchers tested a strong combination of chemotherapy drugs followed by a stem cell transplant in 75 people with aggressive B-cell lymphoma that had spread to the brain or spinal cord. After one year, 58% were cancer-free.
The Lancet. Haematology · NCT02329080
Researchers tested an 8- to 12-week course of glecaprevir/pibrentasvir in 100 Brazilian adults who had never been treated for hepatitis C. Nearly all patients cleared the virus, with few side effects.
Annals of hepatology · NCT03219216
CheckMate 743 tested nivolumab plus ipilimumab — two immunotherapy infusions — against standard chemotherapy in 605 people with untreated unresectable malignant pleural mesothelioma. The combination extended overall survival.
The Lancet · NCT02899299
Patients with lung scarring and high blood pressure in their lungs took an inhaled medicine four times a day. After 16 weeks, they could walk about 100 feet farther in a 6-minute test compared to those who got a placebo. Their heart stress markers also improved.
The New England journal of medicine · NCT02630316
Researchers tested a new automated insulin pump against an existing system in 113 teens and young adults with type 1 diabetes. The newer system reduced high blood sugar episodes by 3% without increasing dangerous low blood sugar.
Lancet (London, England) · NCT03040414
Scientists tested a hepatitis B vaccine in people with chronic lymphocytic leukemia (CLL). They found that patients taking ibrutinib or similar drugs had much lower response rates to the vaccine than those not on treatment. Only 4% of patients on these targeted therapies developed protective antibodies, compared to 28% of untreated patients.
Blood · NCT03685708
Scientists tested whether taking rimegepant every other day could prevent migraines. After three months, people taking the medication had about one fewer migraine day per month compared to those taking a sugar pill.
Lancet (London, England) · NCT03732638
Researchers tested tradipitant, a pill that blocks a receptor involved in nausea, in 152 people with gastroparesis. After 4 weeks, those taking tradipitant had significantly less nausea and more nausea-free days compared to those taking a placebo.
Gastroenterology · NCT02970968
Adults with moderate to severe asthma who weren't doing well on two drugs tried a single inhaler combining three medicines. After 24 weeks, the triple combo improved breathing capacity significantly more than the two-drug version, though it didn't reduce asthma attacks as much as hoped.
The Lancet. Respiratory medicine · NCT02924688
Scientists tested a gene therapy called nadofaragene firadenovec in people whose bladder cancer didn't respond to BCG treatment. About half of patients saw their cancer disappear within three months, and nearly half of those stayed cancer-free for a year.
The Lancet. Oncology · NCT02773849
This long-term extension followed 211 people with hereditary transthyretin amyloidosis who continued patisiran, an IV RNA-silencing infusion, for up to a year. People who started on patisiran kept their nerve function steady; those who switched from inactive comparison began to improve.
The Lancet Neurology · NCT02510261
AFFIRM-AHF tested intravenous ferric carboxymaltose — an iron infusion — in 1,132 people who had been hospitalized for heart failure with low iron stores. Iron repletion cut heart failure hospitalizations over the next year.
The Lancet · NCT02937454
Researchers tested pembrolizumab, an immune therapy, against standard chemotherapy in 307 people with a specific type of advanced colorectal cancer (MSI-H or dMMR). Pembrolizumab doubled the time before cancer worsened compared to chemotherapy, with fewer serious side effects.
The New England journal of medicine · NCT02563002
Researchers tested a hybrid procedure combining heart surface and inside catheter ablation versus standard catheter ablation alone for persistent atrial fibrillation. The combined approach kept 68% of patients free from irregular heart rhythm at one year, compared to 50% with catheter ablation alone.
Circulation. Arrhythmia and electrophysiology · NCT01984346
This trial tested setmelanotide, a daily injection, in people with severe early-onset obesity caused by rare POMC or LEPR gene defects. About 8 in 10 with POMC deficiency and roughly half with LEPR deficiency lost at least 10% of their body weight.
The Lancet Diabetes & Endocrinology · NCT02896192
256 people with bronchiectasis took brensocatib, a pill that blocks an enzyme linked to lung inflammation. Those on the pill went twice as long before their next flare-up compared to people on placebo.
The New England journal of medicine · NCT03218917
Researchers tested golimumab, an antibody drug, in young people just diagnosed with type 1 diabetes. After one year, those taking golimumab made more of their own insulin and needed less injected insulin than those on a dummy treatment.
The New England journal of medicine · NCT02846545
Researchers tested whether adding selinexor to a standard treatment could help people with multiple myeloma who had tried one to three other treatments. Patients who got selinexor with bortezomib and dexamethasone went nearly 14 months before their cancer worsened, compared to about 9.5 months with bortezomib and dexamethasone alone.
Lancet (London, England) · NCT03110562
Scientists tested tazemetostat in 62 people with advanced epithelioid sarcoma, a rare and aggressive soft-tissue cancer. About 15% of patients saw their tumors shrink, and the treatment was generally well tolerated with manageable side effects.
The Lancet. Oncology · NCT02601950
Researchers tested upadacitinib, a pill taken once daily, against abatacept (an IV medication) in 612 people with rheumatoid arthritis who hadn't improved on other treatments. After 12 weeks, upadacitinib reduced disease activity more and helped 30% of patients reach remission compared to 13% with abatacept.
The New England journal of medicine · NCT03086343
Nearly 930 people with relapsing multiple sclerosis were randomly assigned to monthly ofatumumab injections or a daily teriflunomide pill. People taking ofatumumab had nearly half the relapse rate and dramatically fewer new brain lesions on MRI — confirming what the companion ASCLEPIOS II trial found at the same time.
New England Journal of Medicine · NCT02681594
955 people with relapsing multiple sclerosis were randomly assigned to monthly ofatumumab injections or a daily teriflunomide pill. After about 18 months, people taking ofatumumab had half the relapse rate and nearly no new brain lesions on MRI — results that matched its companion trial, ASCLEPIOS I, almost exactly.
New England Journal of Medicine · NCT02792231
Researchers tested whether continuing avelumab immunotherapy after chemotherapy could help people with advanced bladder cancer live longer. Patients who received avelumab lived a median of 21.4 months compared to 14.3 months for those who stopped treatment after chemotherapy.
The New England journal of medicine · NCT02603432
BLISS-LN tested belimumab, a monthly IV antibody, added to standard immune-suppressing therapy in 448 adults with active lupus kidney disease. After two years, more patients on belimumab had improved kidney function and fewer had a kidney flare or died.
New England Journal of Medicine · NCT01639339
EXPLORER-HCM tested mavacamten, a once-daily pill that quiets an overactive heart muscle, in 251 adults with symptomatic obstructive hypertrophic cardiomyopathy. Before this drug, treatment options for the condition were decades old. Patients on mavacamten reported less shortness of breath and could exercise longer.
The Lancet · NCT03470545
Researchers tested a combination of two compounds, sodium phenylbutyrate and taurursodiol (called AMX0035), in 137 people with ALS. Over 24 weeks, people taking AMX0035 had slower decline in daily function compared to those on a dummy treatment.
The New England journal of medicine · NCT03127514
ROAR tested dabrafenib plus trametinib — two daily targeted pills — in 43 people with advanced BRAF-mutant biliary tract cancer who had failed prior chemotherapy. Tumors shrank in about half.
The Lancet Oncology · NCT02034110
Researchers tested ozanimod, a pill taken once daily, in 69 people with moderate to severe Crohn's disease. After 12 weeks, about 4 in 10 patients had their symptoms go into remission, and more than half saw meaningful improvement in daily symptoms like stomach pain and bathroom frequency.
The lancet. Gastroenterology & hepatology · NCT02531113
Scientists tested a one-time gene therapy in four young boys with Duchenne muscular dystrophy. After treatment, all four boys showed muscle improvement that lasted at least a year, with 81% of muscle fibers producing the missing protein.
JAMA neurology · NCT03375164
ASCLEPIOS I and II tested ofatumumab, a monthly self-injection that depletes B cells, against teriflunomide, the leading oral MS pill. Across 1,882 people with relapsing MS, ofatumumab cut the average yearly relapse rate roughly in half.
New England Journal of Medicine · NCT02792218
Researchers compared two blood pressure medicines in older adults with mild memory problems. After one year, people taking candesartan did better on memory and thinking tests than those taking lisinopril, even though both groups had similar blood pressure control.
JAMA network open · NCT01984164
This 16-boy trial tested viltolarsen — a weekly IV infusion that helps the body skip a faulty section of the dystrophin gene — in boys with DMD whose mutation can be treated by exon-53 skipping. Both doses raised dystrophin levels and improved timed motor tests vs. natural history.
JAMA Neurology · NCT02740972
Doctors tested a gentler chemotherapy regimen using treosulfan before cord blood transplants in 130 patients with blood cancers. Most patients' transplants succeeded, with 66% alive at three years. Even patients who had failed a previous transplant saw benefit.
Blood advances · NCT00796068
THALES tested whether adding ticagrelor to aspirin in the first 24 hours after a mild stroke or TIA prevents another stroke. Across 11,016 patients followed for 30 days, the combination cut the risk of a second stroke or death — but caused more serious bleeding.
New England Journal of Medicine · NCT03354429
Scientists tested tofersen, a treatment given by spinal injection, in 50 adults with ALS caused by a specific gene mutation (SOD1). The highest dose reduced a harmful protein in spinal fluid by 33% after 12 weeks, suggesting the drug reached its target.
The New England journal of medicine · NCT02623699
Researchers tested givosiran, a monthly injection that blocks a liver enzyme, in people with acute intermittent porphyria who were having repeated attacks. Patients who got givosiran had 74% fewer porphyria attacks over six months compared to those who got a placebo.
The New England journal of medicine · NCT03338816
Researchers tested relugolix, a daily pill, against leuprolide injections in men with advanced prostate cancer. Relugolix lowered testosterone faster and kept it lower through 48 weeks. Men taking relugolix also had 54% fewer serious heart problems.
The New England journal of medicine · NCT03085095
DEEP-2 compared deferiprone with deferasirox — two oral iron chelators — in 393 children with transfusion-dependent thalassemia. After a year, deferiprone matched deferasirox for keeping iron stores under control.
The Lancet Haematology · NCT01825512
COMBAT tested motixafortide (BL-8040) — a CXCR4-blocking injection — plus pembrolizumab and chemotherapy in 80 people with metastatic pancreatic cancer. The triple combination produced a 32% tumor response rate in the second-line setting.
Nature Medicine · NCT02826486
Researchers tested olaparib in men with advanced prostate cancer that had DNA repair gene changes and stopped responding to hormone therapy. Men taking olaparib went twice as long without their cancer worsening compared to continuing hormone therapy.
The New England journal of medicine · NCT02987543
Researchers tested MOR202, an antibody that targets CD38 on myeloma cells, in 91 people whose cancer had returned or stopped responding to treatment. The drug could be given safely in 30-minute infusions and worked best when combined with other myeloma drugs.
The Lancet. Haematology · NCT01421186
Scientists tested zilucoplan, a self-injected drug that blocks part of the immune system, in 44 people with moderate to severe myasthenia gravis. After 12 weeks, those taking the higher dose had meaningful improvements in muscle strength and daily activities compared to those on placebo.
JAMA neurology · NCT03315130
Researchers tested whether daratumumab given as a shot under the skin worked as well as the standard IV infusion in people with multiple myeloma who had tried at least three other treatments. The shot worked just as well and caused fewer side effects during administration.
The Lancet. Haematology · NCT03277105
This 31-patient trial tested bendamustine — a monthly IV chemotherapy — with weekly dexamethasone in people whose AL amyloidosis came back or did not respond to prior treatment. About 57% had a meaningful blood-test response.
Journal of Clinical Oncology · NCT01222260
Scientists tested daratumumab, an antibody treatment already approved for multiple myeloma, in 22 patients whose AL amyloidosis had come back after prior treatment. The study found that 86% of patients had their disease markers drop significantly, with many seeing kidney and heart function improve.
Blood · NCT02841033
Researchers tested whether heart surgery or stents helped people with both advanced kidney disease and blocked heart arteries. After two years, patients who had surgery were no less likely to die or have a heart attack than those treated with medication alone. Surgery carried higher stroke risk.
The New England journal of medicine · NCT01985360
Researchers tested pembrolizumab (a checkpoint inhibitor) combined with bevacizumab (a blood vessel blocker) in 48 people with metastatic kidney cancer who hadn't received treatment yet. About 61% of patients saw their tumors shrink, and half remained progression-free for over 20 months.
Journal of clinical oncology : official journal of the American Society of Clinical Oncology · NCT02348008
Scientists tested fenfluramine in 87 children with Dravet syndrome who still had frequent seizures despite taking other medications including stiripentol. Children taking fenfluramine had 54% fewer seizures per month than those taking a sugar pill.
JAMA neurology · NCT02926898
Researchers tested an 8-week course of glecaprevir/pibrentasvir in 343 adults with hepatitis C and compensated cirrhosis who had never been treated before. Nearly all patients (98%) were cured across all major virus types, matching the results of a longer 12-week treatment.
Journal of hepatology · NCT03089944
Scientists tested nilotinib, a cancer drug, in 75 people with Parkinson's to see if it could slow the disease. The drug reached the brain and changed several brain chemicals linked to Parkinson's, including reducing harmful protein clumps.
JAMA neurology · NCT02954978
Researchers tested anifrolumab, a monthly infusion, in 362 adults with active lupus. After one year, 48% of people taking anifrolumab had meaningful improvement in their disease compared to 32% taking a dummy treatment.
The New England journal of medicine · NCT02446899
Researchers tested pamrevlumab, an infusion given every three weeks, in 103 people with idiopathic pulmonary fibrosis, a disease that scars the lungs and makes breathing harder. After a year, those who got the treatment lost 60% less lung function than those on placebo.
The Lancet. Respiratory medicine · NCT01890265
Researchers tested whether low-dose colchicine, a common anti-inflammatory drug, could prevent future heart problems in people who recently had a heart attack. Patients taking colchicine daily had 23% fewer major cardiac events compared to those on placebo over nearly two years.
The New England journal of medicine · NCT02551094
This 53-patient trial tested concizumab — a daily under-the-skin injection that boosts the clotting cascade — for hemophilia A and B with or without inhibitors. Bleeding rates dropped substantially across all groups.
Blood · NCT03196284
DAPA-HF tested dapagliflozin, a once-daily diabetes pill, in 4,744 people with heart failure and a weakened pumping heart (reduced ejection fraction). The trial was stopped early after the drug clearly cut hospitalizations and deaths from heart failure.
New England Journal of Medicine · NCT03036124
This 3-patient pilot study gave etranacogene dezaparvovec (AMT-061), a one-time IV gene therapy, to men with severe to moderately severe hemophilia B. Factor IX activity rose to near-normal at 26 weeks, and bleeding stopped completely.
Blood Advances · NCT03489291
ENTRUST-AF PCI tested an edoxaban-based blood thinner regimen against the older warfarin-style regimen in 1,506 people with atrial fibrillation who had heart artery stenting. Edoxaban caused less bleeding without raising clot risk.
The Lancet · NCT02866175
This study tested rogaratinib, a drug that blocks proteins called fibroblast growth factor receptors, in people with advanced cancers who had high levels of these proteins in their tumors. About 15% of patients saw their tumors shrink, including 12 of 52 people with advanced bladder cancer.
The Lancet. Oncology · NCT01976741
Researchers tested whether adding carboplatin to cabazitaxel chemotherapy could help men with advanced prostate cancer that no longer responds to hormone therapy. The combination delayed disease progression by nearly three months compared to cabazitaxel alone, though side effects were more common.
The Lancet. Oncology · NCT01505868
Researchers compared two heart failure drugs in 464 patients with weak heart pumps. After 12 weeks, sacubitril-valsartan didn't reduce artery stiffness more than enalapril, but it did shrink the heart's chambers and improve filling pressure better.
JAMA · NCT02874794
Scientists tested SAGE-217, a pill that works on brain chemicals involved in mood, in people with major depression. After two weeks of daily treatment, depression symptoms dropped significantly more than with a sugar pill.
The New England journal of medicine · NCT03000530
This 92-patient trial gave surgeons 3-D virtual reality models of patient kidneys before robotic partial nephrectomy. Operations done with VR planning had shorter operating times, less blood loss, and shorter hospital stays.
JAMA Network Open · NCT03334344
SUSTAIN-1 tested whether continuing esketamine nasal spray prevents depression from coming back in adults with treatment-resistant depression who initially responded. Among 297 patients in stable remission, those who continued esketamine had about half the relapse rate of those who switched to placebo.
JAMA Psychiatry · NCT02493868
Scientists tested whether pembrolizumab, an immunotherapy drug, was safe for people living with HIV who also had advanced cancer. The study included 30 patients on HIV medications with various cancer types. Pembrolizumab was generally safe when combined with HIV treatment, and some patients' tumors shrank or stopped growing.
JAMA oncology · NCT02595866
Researchers tested selinexor combined with dexamethasone in patients whose myeloma had stopped responding to standard treatments. About 1 in 4 patients saw their cancer shrink, though responses typically lasted only a few months.
The New England journal of medicine · NCT02336815
HOPE tested voxelotor, a daily pill that prevents sickled hemoglobin from clumping, in 274 people with sickle cell disease. After 24 weeks, the higher dose raised hemoglobin levels and reduced markers of red-cell breakdown.
New England Journal of Medicine · NCT03036813
TRANSFORM-2 tested esketamine, a nasal spray form of ketamine, in 236 adults with major depressive disorder that hadn't responded to at least two prior antidepressants. After four weeks of esketamine alongside a newly started oral antidepressant, depression scores improved more than with the antidepressant alone — and many patients felt better within the first 24 hours.
American Journal of Psychiatry · NCT02418585
Researchers tested filgotinib in people with rheumatoid arthritis who hadn't improved with other treatments. After 12 weeks, 66% of patients taking the higher dose had significant symptom improvement, compared to 31% taking a placebo.
JAMA · NCT02873936
This 21-patient pilot study was the first test of mavacamten — a daily oral myosin inhibitor pill — in symptomatic obstructive hypertrophic cardiomyopathy. Heart-blockage pressures fell sharply, exercise capacity rose, and breathing symptoms improved.
Annals of Internal Medicine · NCT02842242
Researchers tested tagraxofusp, a targeted treatment, in 47 adults with blastic plasmacytoid dendritic-cell neoplasm (BPDCN), a rare and aggressive blood cancer. In previously untreated patients, 72% achieved complete or near-complete remission, and 45% were able to proceed to stem-cell transplant.
The New England journal of medicine · NCT02113982
MyPathway tested two HER2-targeting antibodies — pertuzumab and trastuzumab — in 57 people with HER2-amplified metastatic colorectal cancer that had failed standard chemotherapy. About a third saw their tumors shrink.
The Lancet Oncology · NCT02091141
Researchers tested luspatercept in 64 people with beta-thalassemia, a blood disorder that causes anemia. Among those needing regular transfusions, 81% needed at least 20% fewer transfusions. Among those not needing regular transfusions, 58% saw their hemoglobin levels rise by at least 1.5 g/dL.
Blood · NCT01749540
Scientists tested luspatercept in 64 people with beta-thalassemia, a blood disorder that causes anemia. Among those who needed regular transfusions, 81% reduced their transfusion needs by at least 20%. Among those who didn't need regular transfusions, 58% saw meaningful increases in hemoglobin levels.
Blood · NCT02268409
This first-in-human study tested tisotumab vedotin — an antibody-drug conjugate targeting tissue factor on tumors — in 147 people with various advanced cancers. Tumors shrank in about 16% across the mix; the drug went on to win FDA approval in cervical cancer.
The Lancet Oncology · NCT02001623
Researchers compared two ways to harvest veins for heart bypass surgery: open surgery (cutting the leg) versus endoscopic (small incisions with a camera). After nearly 3 years, both methods led to similar rates of death, heart attacks, and repeat procedures.
The New England journal of medicine · NCT01850082
This early study tested elexacaftor-tezacaftor-ivacaftor — the triple-combination pill now sold as Trikafta — in 225 people with cystic fibrosis carrying at least one Phe508del mutation. Lung function improved sharply within weeks, paving the way for the Phase 3 trials that led to FDA approval.
New England Journal of Medicine · NCT03227471
Scientists tested a three-drug combination (VX-659, tezacaftor, and ivacaftor) in people with cystic fibrosis caused by specific genetic mutations. Patients saw significant improvements in lung function—up to 13 percentage points in some groups—with mild to moderate side effects.
The New England journal of medicine · NCT03224351
Researchers tested BMS-986165, a pill that blocks a protein called TYK2 involved in psoriasis inflammation. After 12 weeks, 75% of patients taking the highest dose saw at least three-quarters of their skin clear up, compared to only 7% on placebo.
The New England journal of medicine · NCT02931838
UltIMMa-1 and UltIMMa-2 tested risankizumab, an IL-23-blocking antibody given as an injection every 12 weeks, against ustekinumab and placebo in 997 adults with moderate-to-severe plaque psoriasis.
The Lancet · NCT02684370
Researchers tested 20 children and adults with sickle cell disease using heart imaging and exercise tests. Nearly all participants had reduced exercise capacity, and those with a heart pumping problem called diastolic dysfunction showed the greatest exercise limitations.
Pediatric blood & cancer · NCT02410811
mSToPS tested whether a 2-week home-worn ECG patch helps detect undiagnosed atrial fibrillation in 2,659 high-risk adults. Immediate monitoring caught 4x more new AFib cases at 4 months than waiting.
JAMA · NCT02506244
Researchers tested ivacaftor in babies aged 12 to 24 months with cystic fibrosis and a specific gene mutation. The medicine was safe and worked well, lowering sweat chloride levels (a key marker of the disease) and showing early signs of protecting pancreas function.
The Lancet. Respiratory medicine · NCT02725567
This early study gave 26 veterans, firefighters, and police officers with chronic PTSD either an active dose of MDMA (75 or 125 mg) or a low control dose during therapy sessions. PTSD symptoms dropped sharply at the active doses, and the gains held a year later.
The Lancet Psychiatry · NCT01211405
KEYNOTE-189 tested pembrolizumab, an immunotherapy that releases brakes on the immune system, added to standard chemotherapy as the first treatment for 616 people with metastatic non-squamous non-small cell lung cancer without targetable mutations.
New England Journal of Medicine · NCT02578680
This 70-child trial tested monthly oral vitamin D vs. low-dose vitamin D in children with sickle cell disease. After two years, both doses cut respiratory illness rates by more than 50%.
Blood Advances · NCT01443728
COLUMBUS tested encorafenib plus binimetinib — two daily targeted pills — against vemurafenib alone in 577 people with advanced BRAF-mutant melanoma. The combination doubled the time before the cancer progressed.
The Lancet Oncology · NCT01909453
Scientists tested sirolimus in 40 people with active lupus who hadn't responded to standard treatments. After 12 months, more than half showed meaningful improvement in their symptoms and were able to reduce their steroid doses by two-thirds.
Lancet (London, England) · NCT00779194
Researchers tested a one-time gene therapy called AMT-060 in 10 adults with severe hemophilia B. After treatment, the body made its own clotting factor IX. Spontaneous bleeding dropped by 53% to 70%, and most patients stopped needing regular clotting factor infusions.
Blood · NCT02396342
DESSOLVE III compared the MiStent (a sirolimus-eluting stent with a fully absorbable polymer coating) with the Xience stent (a leading durable polymer stent) in 1,398 patients having heart artery stenting. At one year, the two performed equally well on heart events and stent safety.
The Lancet · NCT02385279
Scientists tested tisagenlecleucel, a therapy that reprograms a patient's own immune cells to fight cancer, in 75 children and young adults with leukemia that had come back or wasn't responding to treatment. Within three months, 81% of patients had no detectable cancer, and many stayed in remission for over a year.
The New England journal of medicine · NCT02435849
Researchers tested high-dose testosterone injections in men with advanced prostate cancer that stopped responding to enzalutamide. This cycling approach helped 30% of men and also made the cancer respond again to enzalutamide in most who tried it later.
The Lancet. Oncology · NCT02090114
Researchers tested a one-time gene therapy infusion in nine men with severe hemophilia A. Six of seven people who got the highest dose reached normal clotting factor levels that lasted a full year, and bleeding events dropped dramatically.
The New England journal of medicine · NCT02576795
Researchers tested a one-time gene therapy in 10 men with hemophilia B. After treatment, 9 of 10 patients stopped having bleeding episodes, and 8 of 10 no longer needed clotting factor infusions.
The New England journal of medicine · NCT02484092
This 23-patient study tested partially HLA-mismatched unrelated donor bone marrow transplant with high-dose post-transplant cyclophosphamide for blood cancers. There were no graft failures or severe acute graft-versus-host disease, and 1-year survival was 75%.
Blood Advances · NCT01203722
Researchers tested a non-invasive brain procedure using focused ultrasound waves to calm tremors in Parkinson's patients whose medications had stopped working. The treatment reduced hand tremors by 62% compared to 22% in patients who received a fake procedure.
JAMA neurology · NCT01772693
Researchers tested whether taking lenalidomide after completing second-line treatment could keep chronic lymphocytic leukemia (CLL) from coming back. Patients on lenalidomide went nearly three years before their cancer progressed, compared to nine months for those on a sugar pill.
The Lancet. Haematology · NCT00774345
Researchers tested combining two immunotherapy drugs—pembrolizumab at full dose with a lower dose of ipilimumab—in 153 people with advanced melanoma. 61% of patients saw their tumors shrink, with manageable side effects.
The Lancet. Oncology · NCT02089685
Researchers tested selinexor, a cancer drug, in 192 patients and discovered why it causes dangerous drops in platelet counts. They found the drug blocks a signal that helps the body make platelets, but giving patients a break from the drug and using platelet-boosting medications can reverse the problem.
Blood · NCT01607905
Researchers tested whether treating all blocked arteries during a heart attack—guided by blood flow measurements—works better than treating only the main blockage. Patients who got complete treatment had 65% fewer heart problems over the next year.
The New England journal of medicine · NCT01399736
Researchers tested a gene therapy that adds a modified gene to a patient's own blood stem cells. Fifteen months after treatment, the patient had no sickle cell crises and the therapy gene made up about half of their hemoglobin.
The New England journal of medicine · NCT02151526
419 people with EGFR-mutant non-small cell lung cancer whose tumors had developed a specific resistance mutation (T790M) were assigned to osimertinib or platinum-based chemotherapy. Osimertinib more than doubled progression-free survival and improved overall survival, with fewer severe side effects than chemotherapy.
New England Journal of Medicine · NCT02151981
Researchers tested atezolizumab, an immunotherapy, in 119 people with advanced bladder cancer who couldn't receive standard chemotherapy. Nearly a quarter of patients responded to treatment, and about half of those responses lasted throughout the study.
Lancet (London, England) · NCT02807636
This early study gave nusinersen — an injection into the spinal fluid that boosts motor neuron protein — to 20 infants with SMA type 1, the most severe form. Babies hit motor milestones they would not have reached without treatment, and survival improved compared with natural history.
The Lancet · NCT02386553
This study tested pembrolizumab (Keytruda) in patients with melanoma or lung cancer that had spread to the brain. About one in four to one in three patients saw their brain tumors shrink, and most responses lasted through the study period.
The Lancet. Oncology · NCT02085070
Researchers tested ozanimod, a pill that reduces immune cells traveling to the gut, in 197 adults with moderate-to-severe ulcerative colitis. After 8 weeks, 16% of patients taking the 1 mg dose achieved remission compared to 6% taking placebo.
The New England journal of medicine · NCT01647516
Researchers tested a gentler chemotherapy combination called EPOCH-R in 30 adults with Burkitt's lymphoma, including people with HIV. After 6–7 years of follow-up, 95–100% of patients were alive and cancer-free, with fewer serious side effects than standard intensive treatment.
The New England journal of medicine · NCT00006436
This early study tested combining two immunotherapy drugs—nivolumab and ipilimumab—in people with advanced melanoma. Over half of patients who received both drugs together saw their tumors shrink by at least 80%.
The New England journal of medicine · NCT01024231
This study tested combining two iron-removal medications—deferasirox (taken daily by mouth) and deferoxamine (given by infusion 3–7 days a week)—in 22 thalassemia patients with dangerous iron buildup. After one year, liver iron dropped by 31% and all six patients with heart iron overload showed improvement.
Blood cells, molecules & diseases · NCT00901199
Researchers tested whether hydroxyurea could prevent organ damage in very young children (9–18 months old) with sickle cell disease. While the drug didn't stop spleen or kidney problems, it cut painful episodes in half and dramatically reduced hand-foot syndrome.
Lancet (London, England) · NCT00006400
This study tested pazopanib, a daily oral pill that blocks blood vessel growth in tumors, in 39 people with advanced radioiodine-resistant differentiated thyroid cancer. Tumors shrank meaningfully in about half.
The Lancet Oncology · NCT00625846
Scientists tested whether deferasirox could remove iron buildup from the hearts of 28 people with beta-thalassemia. After 18 months, the drug helped patients with mild to moderate iron overload but didn't work well for those with severe iron buildup in the liver.
Blood · NCT00447694
This trial compared ethosuximide, valproic acid, and lamotrigine in 453 children with newly diagnosed absence epilepsy. After 16 weeks, ethosuximide and valproic acid both stopped seizures far more often than lamotrigine, and ethosuximide caused fewer attention problems than valproic acid.
New England Journal of Medicine · NCT00088452
This study tested whether adding zoledronic acid (a bone-strengthening drug) to standard hormone therapy could help premenopausal women with hormone-responsive early breast cancer. After 4 years, women who received zoledronic acid had a 36% lower chance of their cancer coming back compared to those on hormone therapy alone.
The New England journal of medicine · NCT00295646