Myelodysplastic syndromes are a group of bone marrow disorders where blood cells don't develop properly, leading to low counts and a risk of progression to acute leukemia. Treatment ranges from supportive care and growth factors to drugs like azacitidine and lenalidomide, and stem cell transplant for younger or higher-risk patients. Many people live for years with stable disease.
What's actually going on in research
Trials are testing venetoclax combinations that borrow strategies from acute leukemia treatment, oral azacitidine to extend response after initial therapy, and drugs targeting specific mutations like TP53 and splicing factor genes. Researchers are also studying immune checkpoint inhibitors, CAR-T cells adapted for MDS, and ways to predict who will respond to existing drugs.
Venetoclax combinations
This pill blocks a protein that keeps cancer cells alive and is FDA-approved for some leukemias. Trials are pairing it with azacitidine or decitabine to see if the combination works better than either drug alone in higher-risk MDS.
Mutation-targeted drugs
Drugs aimed at TP53 mutations, common in hard-to-treat MDS, are in testing. Other trials target splicing factor mutations, which drive many cases of MDS and have no specific treatment yet.
Oral azacitidine maintenance
An oral form of azacitidine, taken daily at home, is FDA-approved to extend remission after initial treatment. Trials are exploring whether starting it earlier or combining it with other drugs improves outcomes.
What to know before you search
Eligibility often depends on MDS subtype, risk score, blood counts, genetic mutations, prior treatments, and whether you're a transplant candidate.
What types of trials are currently open
- Treatment trials — Testing new drug combinations or next-generation versions of azacitidine and lenalidomide to see if they improve blood counts or delay progression to leukemia.
- Transplant trials — Studies refining when to transplant, what conditioning regimen to use, and how to manage complications afterward.
- Maintenance trials — Testing whether drugs taken after initial treatment can keep MDS under control longer.
- Mutation-based trials — Enrolling people based on genetic features of their MDS cells to test drugs that target those specific mutations.
- Observational studies — Following people with MDS over time to learn how the disease changes, what predicts transformation to leukemia, and who benefits from which treatments.
Recently added Myelodysplastic Syndromes trials
Receive antifungal medication to prevent serious infections during cancer treatment
This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (50 mg/day, intravenous, once daily) for the prevention of invasive fungal infections in adult patients (aged 18-75 years) with hematological malignancies who develop prolonged neutropenia (absolute neutrophil count ≤0.5×10\^9/L, expected to last \>7 days) and are at high risk for invasive fungal disease. Participants are those who, per the treating physician's routine clinical decision, have been initiated on liposomal amphotericin B prophylaxis at 50 mg/day due to intolerance or toxicity to other antifungal agents. The primary outcome is the incidence of proven or probable invasive fungal disease. Secondary outcomes include incidence of pneumonia, persistent unexplained fever \>4 days, use of additional systemic antifungal therapy, and adverse events. A total of 30 participants will be enrolled. Data will be collected at baseline, during treatment, and within 7 days after treatment completion.
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Phase I Study Evaluating Safety, Tolerability, PK and PD of Single and Multiple Doses of TUL321 Capsule in Healthy Participants and Food Effect on PK
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