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 70 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06712810RecruitingPhase 1
Q702 for blood cancers and histiocytic disorders
This study tests an experimental pill called Q702 for people with certain blood cancers and related disorders (like histiocytoses, myelofibrosis, and lymphomas) that have not responded to standard treatments. It aims to see if Q702 is safe and effective.
Scottsdale, ArizonaAges 18 years+ - NCT06802315RecruitingPhase 2
Radiation therapy before stem cell transplant for high-risk blood cancers
This trial tests a precise type of radiation called intensity modulated total marrow irradiation before a stem cell transplant. It aims to destroy cancer cells while reducing side effects for patients with high-risk leukemia or myelodysplastic syndrome.
Chicago, IllinoisAges 18–65 - NCT06928662RecruitingPhase 1/Phase 2
Chemo, radiation, and donor stem cell transplant for high-risk blood cancers
This trial tests a strong chemotherapy and radiation combo followed by a donor stem cell transplant for people with aggressive blood cancers (like AML or MDS) that haven't responded to earlier treatments or came back. It aims to help prevent the cancer from returning.
Seattle, WashingtonAges 18 years+ - NCT06994676RecruitingPhase 1
Study of CBX-250 for advanced myeloid leukemias
This study tests an experimental drug, CBX-250, for people with certain advanced blood cancers (like AML) that have not responded to standard treatments or have returned. The drug works only for people who have a specific genetic marker (HLA-A*02:01). The goal is to see if it is safe and how well it works.
Duarte, CaliforniaAges 12 years+ - NCT07107126RecruitingPhase 1
Testing a new drug for relapsed AML or high-risk MDS
This trial tests a new drug called RPT1G for adults with acute myeloid leukemia or high-risk myelodysplastic syndromes that have not responded to standard treatments. The goal is to see if the drug is safe and if it shows signs of helping control the disease.
New York, New YorkAges 18 years+ - 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+ - 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+ - 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 - 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 - 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+ - 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 - 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 - 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
Axatilimab with or without azacitidine for advanced blood cancers
This trial tests a new drug, axatilimab, given alone or with another drug (azacitidine) for people with advanced forms of blood cancers like certain leukemias and myelofibrosis. It may help if your disease has not responded to earlier treatments or if you are starting treatment for the first time.
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 - NCT02508038RecruitingPhase 1
Test CD19+ depleted donor transplant for hard childhood cancers
This Phase 1 study tests a special type of half-matched (haploidentical) stem cell transplant plus medicines, aiming to reduce cancer relapse after transplant. It may help children and teens with certain leukemia/lymphoma or specific solid tumors who do not have a fully matched donor or whose best options are limited.
Madison, WisconsinAges 7 months–21 years - NCT05211570RecruitingPhase 1/Phase 2
Study drug AB8939 for AML after prior treatments
This Phase 1/2 study tests AB8939 in people with acute myeloid leukemia (AML) whose disease has come back or did not respond to earlier treatment. It may help researchers find a safe dose and learn whether the treatment can control the leukemia.
Houston, TexasAges 18 years+ - NCT04187703RecruitingEarly Phase 1
Testing azacitidine and decitabine for bone marrow cancers
This early-phase study tests whether combining two “epigenetic” medicines—azacitidine and decitabine—can improve outcomes for certain bone marrow cancers. It may help if you have a type of myelodysplastic syndrome (MDS) or MDS/MPN overlap that is expected to be sensitive to these medicines.
Cleveland, OhioAges 18 years+
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Common questions
- Are there clinical trials for refractory high risk myelodysplastic syndrome?
- Yes. Clin2 currently lists 70 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.