Myelodysplastic syndromes are a group of bone marrow disorders where the marrow makes blood cells that don't develop properly, leading to low counts and risk of progression to leukemia. About 10,000 people in the U.S. are diagnosed each year, most over age 65. Current treatments include supportive care, hypomethylating agents like azacitidine and decitabine, and stem cell transplant for eligible patients.
What's actually going on in research
Trials are testing oral hypomethylating agents that avoid the need for injections, combinations of hypomethylating agents with venetoclax or other targeted drugs, telomerase inhibitors like imetelstat for lower-risk disease, and newer approaches to preparing patients for transplant. Researchers are also studying genetic mutations that predict which treatments will work and whether early intervention can delay progression.
Oral hypomethylating agents
Oral versions of azacitidine are now FDA-approved, allowing treatment at home instead of weekly clinic visits. Oral decitabine combined with cedazuridine (Inqovi) offers similar convenience for certain patients.
Venetoclax combinations
Adding venetoclax, a drug that targets a survival protein in cancer cells, to azacitidine is being tested to see if it improves responses. Early data suggest the combination may work for some patients who don't respond to azacitidine alone.
Telomerase inhibition
Imetelstat blocks an enzyme that helps cells replicate indefinitely, and has shown promise in reducing transfusion needs for lower-risk MDS. It's being studied as an option for patients who rely on frequent transfusions.
What to know before you search
Eligibility typically depends on MDS subtype, risk score, blast percentage, blood counts, prior treatments, and whether you're a candidate for transplant.
What types of trials are currently open
- Treatment trials — Testing new drugs or drug combinations against current standards like azacitidine, often for higher-risk MDS.
- Lower-risk trials — Studies of drugs that reduce transfusion needs or improve blood counts in people with lower-risk disease who don't need immediate transplant.
- Transplant trials — Testing new conditioning regimens or post-transplant strategies to reduce complications and relapse risk.
- Maintenance trials — Studies of drugs given after transplant or initial treatment to prevent relapse.
- Biomarker studies — Following patients to understand how genetic mutations and other markers predict disease behavior and treatment response.
Recently added Myelodysplastic Syndromes trials
Share medical data and samples to advance trisomy 8 research
Background: Trisomy 8 mosaicism is a genetic disorder that can increase inflammation in the body. Symptoms include fevers; sores or ulcers in the mouth, digestive tract, or genital area; skin rashes; problems in organs or tissues; and changes in bone marrow cells. Researchers want to conduct a natural history study to learn more about these symptoms and what causes them. Objective: To gather data and samples from people with and without the trisomy 8 mosaicism. Eligibility: People of any age with the trisomy 8 gene mosaicism. Their healthy relatives are also needed. Design: Affected participants will have visits every 1 to 2 years for 30 years at NIH. Each visit will take 1 to 5 days and may be in-person or remote. With remote visits, participants may have a video call with the study team and samples may be sent to researchers by mail. Participants may have these procedures: Physical exam, with blood tests. Tests of brain function and motor skills. Sensory tests. Researchers will see how participants respond to sensations such as pinpricks, heat, cold, and pressure. Magnetic resonance imaging (MRI) scan of the brain and/or spine. X-ray of the spine. Ultrasound test of heart function (echocardiogram). Tissues samples (biopsies) collected from the skin, inside of the mouth, and bone marrow. Swabs to collect cells from the mouth, skin, and vagina. Collection of blood, stool, urine, saliva, hair, and fingernail samples. X-rays, MRI, and heart tests will be done only once. Other procedures may be repeated at each visit. All tests and procedures are voluntary. Healthy relatives who enroll will have a baseline visit and then follow-up visits as needed. They will have a physical exam. The inside of their mouth may be swabbed. Samples of blood, stool, urine, and saliva may be taken.
Caris Chromoseq Data Collection
The study will collect clinical data on patients who receive the Caris Chromoseq assay for an underlying hematologic malignancy. The assay provides risk stratification for patients with acute myeloid leukemia (AML) myelodysplastic syndrome (MDS), or myeloproliferative neoplasms (MPN). The hypothesis of the study is that Caris Chromoseq compares favorably to conventional cytogenetics, FISH, and NGS analysis in terms of risk stratification capabilities, ease of use, and turnaround time.
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