Clinical trials
Refractory High Risk Myelodysplastic Syndrome clinical trials
Below are recruiting refractory high risk myelodysplastic syndrome clinical trials, each written for real people, not researchers. We’re tracking 74 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT03896269RecruitingPhase 1
CPX-351 for high-risk MDS or CMML that came back
This Phase 1 trial studies CPX-351, a cancer medicine, in people with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) whose disease relapsed or did not respond to prior treatment. It may help shrink or control the leukemia and improve blood counts in patients who cannot or do not want a stem cell transplant.
Houston, TexasAges 18 years+ - NCT03630991RecruitingPhase 1
Calcium chelator medicine for AML or MDS during chemotherapy
This Phase 1 study tests whether edetate calcium disodium (also called succimer) can be safely given to people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) while they are on chemotherapy. The goal is to learn the safest dose and whether it has signs of helping the blood cancer respond to treatment.
Houston, TexasAges 1 year+ - NCT03661307RecruitingPhase 1/Phase 2
Study of quizartinib, decitabine, and venetoclax for acute myeloid leukemia or high-risk myelodysplastic syndrome
This trial combines three drugs to treat certain types of leukemia or a related blood condition. It may be an option for people who cannot have standard intensive chemo, or for those whose disease has come back.
Houston, TexasAges 18 years+ - NCT03670966RecruitingPhase 1/Phase 2
Targeted iodine therapy, then donor transplant for hard leukemias
This trial tests an antibody-linked radioactive iodine treatment, followed by a donor stem cell transplant, for people with relapsed or refractory high-risk acute leukemia or certain MDS. It may help patients by targeting leukemia cells and then using a new immune system to control the disease.
Seattle, WashingtonAges 18–75 - NCT03383575RecruitingPhase 2
Azacitidine plus enasidenib for IDH2-mutant MDS
This trial tests whether combining azacitidine with enasidenib helps people with myelodysplastic syndrome (MDS) that has an IDH2 mutation. It’s designed for people who are either new to “hypomethylating” treatment or whose MDS returned/worsened after it.
Baltimore, MarylandAges 12 years+ - NCT03214562RecruitingPhase 1/Phase 2
Venetoclax plus chemotherapy for acute leukemia
This trial studies how well venetoclax combined with chemotherapy treats newly diagnosed or returning (relapsed) or hard-to-treat (refractory) acute myeloid leukemia (AML). It’s mainly for people who can’t use standard AML therapy well, or whose disease came back.
Houston, TexasAges 18 years+ - NCT03128034RecruitingPhase 1/Phase 2
Astatine-211 treatment before stem cell transplant for high-risk blood cancers
This trial tests a targeted radiation drug (Astatine-211) given before a donor stem cell transplant for people with high-risk leukemia or certain bone-marrow cancers. The goal is to better control the cancer before the transplant.
Seattle, WashingtonAges 18–75 - NCT03113643RecruitingPhase 1
SL-401 with standard drugs for hard-to-treat blood cancers
This Phase 1 study tests SL-401, given with azacitidine and sometimes venetoclax, to treat acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (MDS), or blastic plasmacytoid dendritic cell neoplasm (BPDCN). It aims to find a safe dose and see whether the combination can improve blood cancer control.
Duarte, CaliforniaAges 18 years+ - NCT02727803RecruitingPhase 2
Personalized natural killer cell therapy for blood cancers
This trial tests a customized immune-cell treatment (NK cells) for certain blood cancers to try to control or eliminate cancer cells. You may qualify if your cancer has come back, did not fully respond, or has higher risk features.
Houston, TexasAges 15–80 - NCT01515527RecruitingPhase 2
Cladribine plus low-dose cytarabine and decitabine for AML or MDS
This Phase 2 study tests a combination chemotherapy plan (cladribine, low-dose cytarabine, and decitabine) for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). It aims to shrink the leukemia/MDS and improve outcomes, including for certain people who may not tolerate standard treatment.
Houston, TexasAges 60 years+ - NCT02115295RecruitingPhase 2
Venetoclax plus chemotherapy for AML, high-risk MDS, or blast CML
This trial tests a combination treatment (venetoclax plus several chemotherapy medicines) for people with AML, high-risk MDS, or CML that has progressed into a blast phase. It may help by killing leukemia-like blood cells and improving disease control.
Houston, TexasAges 18–65 - NCT00801489RecruitingPhase 2
New treatment for AML or high-risk MDS using combination chemo
This trial tests a combination treatment (chemo drugs plus a growth-factor medicine) for people newly diagnosed with acute myeloid leukemia (AML) or certain high-risk myelodysplastic syndromes (MDS). It may help by targeting cancer cells in the blood and bone marrow and improving remission chances.
Houston, TexasAges 18 years+ - NCT05601726RecruitingPhase 1/Phase 2
Study of a new drug for hard-to-treat AML or high-risk MDS
This early study tests a new medicine (ABD-3001) to see if it’s safe and tolerable, how the body handles it, and whether it shows early signs of helping people with relapsed or hard-to-treat AML or high-risk MDS. It may be an option if standard treatments have already failed and you can’t receive a stem cell transplant.
Marseille, FranceAges 18 years+ - NCT07532824RecruitingPhase 1/Phase 2
Proton Radiation and Stem Cell Transplant for High-Risk Blood Cancers
This trial tests a new type of radiation therapy (proton-based) combined with a stem cell transplant from a donor to treat advanced forms of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The goal is to improve survival in patients with aggressive or hard-to-treat disease.
PragueAges 18–65 - NCT05805605RecruitingPhase 2
Low-intensity stem cell transplant with donor cells and blood-protection
This Phase 2 study tests a low-intensity bone marrow/stem cell transplant using a matched donor plus two medicines to help prevent the donor cells from attacking you. It aims to treat certain blood cancers—especially when standard treatment has not fully worked or when you are in remission but at higher risk.
Minneapolis, MinnesotaAges Up to 75 years - NCT06303193RecruitingPhase 1/Phase 2
Testing a kinase inhibitor drug for MDS and MDS/MPN in adults and teens
This trial tests a drug called pacritinib in people with myelodysplastic syndromes (MDS) or MDS/myeloproliferative neoplasms (MDS/MPN). The drug targets several proteins that may help control the disease, especially for those who have not responded to other treatments.
Bethesda, MarylandAges 12–120 - NCT04140487RecruitingPhase 1/Phase 2
Study drugs for certain FLT3-mutated blood cancers that have returned
This trial tests a combination of three medicines—azacitidine, venetoclax, and gilteritinib—in people with specific gene changes (FLT3 mutations) in leukemia and related bone marrow diseases that have come back or are hard to treat. The goal is to see if this drug combination can control the disease and improve outcomes.
Houston, TexasAges 18 years+ - NCT07523555RecruitingPhase 1/Phase 2
Dual-Target CAR-T Cells for Blood Cancers That Haven't Responded to Treatment
This study tests a new type of cell therapy called CAR-T cells that are designed to target two different proteins on cancer cells. These cells are made from your own immune cells, trained in the lab, and then put back into your body to fight blood cancers that have come back or stopped responding to previous treatments.
Shenzhen, GuangdongAges 18–75 - NCT07237230Recruiting
Gamma-delta T cells to prevent leukemia relapse after transplant
This trial tests if a special type of immune cell called gamma-delta T cells can help prevent acute myeloid leukemia from coming back after a stem cell transplant. It is for people with high-risk features who have recently had a transplant.
Wuhan, HubeiAges 18–65 - NCT06523556RecruitingPhase 1/Phase 2
Study of axatilimab with or without azacitidine for advanced blood cancers
This trial tests a new drug called axatilimab, given alone or with another drug, for people with certain blood cancers that are advanced or hard to treat. The goal is to see if this combination can help control the disease.
Columbus, OhioAges 18 years+ - NCT04708054RecruitingPhase 2/Phase 3
Venetoclax with busulfan chemo for high-risk AML or MDS
This study tests whether adding venetoclax to a specific busulfan-based chemotherapy plan improves outcomes for people with high-risk AML or MDS. It mainly targets patients whose leukemia/marrow disease still has high-risk features or measurable disease after initial treatment.
Houston, TexasAges 18–70 - NCT04167696RecruitingPhase 1
Dose-finding drug study for AML or MDS after standard options fail
This Phase 1 trial tests CYAD-02 to find the safest and recommended dose for people with AML or MDS that has returned or did not respond to prior treatment. It may help if you have limited treatment options, but participation also requires meeting specific heart, lung, and lab safety requirements.
Jacksonville, FloridaAges 18 years+ - NCT03850574RecruitingPhase 1/Phase 2
Tuspetinib for relapsed or hard-to-treat AML
This early-phase study tests tuspetinib in people with AML that has come back or did not respond to treatment. The goal is to learn whether it is safe and tolerable, how the drug behaves in the body, and whether it shows signs of benefit.
Birmingham, AlabamaAges 18 years+ - NCT06013423RecruitingPhase 2
Cord blood transplant with chemo and radiation for high-risk blood cancers
This trial tests a cord blood transplant after a reduced-intensity chemotherapy and radiation regimen for patients with high-risk blood cancers like leukemia, lymphoma, or MDS. The goal is to see if this approach can help destroy cancer cells and restore healthy blood cells.
Seattle, WashingtonAges 6 months–65 years
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Common questions
- Are there clinical trials for refractory high risk myelodysplastic syndrome?
- Yes. Clin2 currently lists 74 recruiting refractory high risk myelodysplastic syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a refractory high risk myelodysplastic syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a refractory high risk myelodysplastic syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.