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
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
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
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
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
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
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
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
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
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
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 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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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 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
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
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
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
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
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
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
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
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
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
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
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 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
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