Clinical trials
Refractory Myelodysplastic Syndrome clinical trials
Below are recruiting refractory myelodysplastic syndrome clinical trials, each written for real people, not researchers. We’re tracking 106 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT02074839RecruitingPhase 1
Oral AG-120 for advanced blood cancers with an IDH1 mutation
This Phase 1 study tests an oral medicine called AG-120 in adults with advanced blood cancers that have a specific IDH1 mutation. It also looks at safety and how the treatment works, with frequent blood, urine, and bone marrow samples.
Birmingham, AlabamaAges 18 years+ - NCT06765876RecruitingEarly Phase 1
CAR T-cell therapy for CD123+ blood cancers
This trial tests a new type of immunotherapy called CAR T-cells that target a protein called CD123 on cancer cells. It is for people with certain blood cancers (AML, MDS-IB2, BPDCN, or ALL) that have come back or not responded to other treatments.
PragueAges 18–70 - NCT01174108RecruitingPhase 2
Stem cell transplant with donor T-cells for severe bone marrow failure
This Phase 2 study tests a specific type of donor stem cell transplant for severe bone marrow failure conditions, aiming to improve cure rates while reducing transplant risks. It includes people with diseases like severe aplastic anemia and certain bone marrow disorders, especially when standard transplant plans feel too risky.
Baltimore, MarylandAges 4–80 - NCT05925504RecruitingPhase 2
Lowering luspatercept dose for lower-risk myelodysplastic anemia
This Phase 2 study tests whether gradually lowering the dose of luspatercept can help people with lower-risk myelodysplastic syndromes (MDS) who have anemia. It may help reduce side effects while still improving blood counts or symptoms.
Tianjin, Tianjin MunicipalityAges 18 years+ - NCT04093570Enrolling by invitationPhase 2
Extension study for people continuing ASTX727 from a prior trial
This study checks whether continuing ASTX727 for people who already benefited from it in an earlier Taiho study is safe and effective. It may be an option if you stayed on ASTX727 at the end of your parent study and your doctor felt it was helping you.
Fountain Valley, CaliforniaAges 18 years+ - NCT04227847RecruitingPhase 1
Study of a CD70-targeting drug for blood cancer
This Phase 1 study tests the safety of SEA-CD70, a drug that targets CD70, in people with certain blood cancers like MDS and AML. It may help doctors learn whether this approach can be used safely for patients whose disease has come back or not responded to standard treatments.
Birmingham, AlabamaAges 18 years+ - NCT03016806Recruiting
Umbilical cord blood transplant from unrelated donors
This trial studies transplanting healthy blood-forming cells from an umbilical cord blood donor who is not related to you. It may help people with certain blood cancers, immune disorders, or other serious diseases when a standard transplant plan is needed.
Rochester, New YorkAges 2 months–75 years - NCT03520647RecruitingPhase 2
Haplo transplant for severe bone marrow failure using donor cells
This trial tests a stem cell transplant from a closely matched but not fully matched family donor (haploidentical), with chemotherapy after the transplant to help prevent graft-versus-host disease (GVHD). It may help people with severe aplastic anemia, certain MDS after transformation, or PNH when other treatments have not worked or are not available.
Bethesda, MarylandAges 4–75 - NCT03533816RecruitingPhase 1
Gamma delta T-cell infusion after stem cell transplant
This early-phase study tests giving a special immune-cell treatment after an allogeneic (donor) stem cell transplant, plus a medication given after transplant to help the new immune system work better. It may help prevent or treat cancer returning after transplant.
Westwood, KansasAges 18–65 - NCT04068597RecruitingPhase 1/Phase 2
Testing inobrodib for blood cancers that came back
This is an early-stage study of inobrodib (CCS1477) for blood cancers like lymphoma, multiple myeloma, or acute myeloid leukemia that have returned or did not respond to standard treatment. It aims to see whether the drug is safe and can help control the cancer.
Atlanta, GeorgiaAges 18 years+ - NCT04275518RecruitingPhase 1
Trial for relapsed leukemia or risky MDS with new drug
This Phase 1 study tests APG-115 alone or with azacitidine or cytarabine in people whose acute myeloid leukemia (AML) or high-risk MDS has come back or hasn’t responded. The goal is to see safety and whether the treatment can work for these conditions.
Beijing, Beijing MunicipalityAges 18 years+ - NCT04581512RecruitingPhase 1/Phase 2
Safety study of EP0042 for certain AML patients
This Phase 1/2 study tests how safe and tolerable EP0042 is, and looks at patients with a specific type of AML that has come back or did not respond to prior treatment. You may be considered if your AML diagnosis is confirmed and your lab tests, overall health, and medication history fit the study rules.
Perth, Western AustraliaAges 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 - NCT04988555RecruitingPhase 1/Phase 2
Study of an investigational drug for high-risk acute leukemia
This Phase 1/2 study tests enzomenib (DSP-5336) in people with relapsed or hard-to-treat acute leukemia (and, in some places, certain other blood cancers). The goal is to see how the drug works and how safe it is, especially for leukemia cases with specific genetic changes (KMT2A/MLL or NPM1).
Newport Beach, CaliforniaAges 12 years+ - NCT05181735RecruitingPhase 1/Phase 2
Testing luspatercept for low-risk MDS with low hemoglobin
This Phase 1/2 study tests luspatercept combined with another approach in people with low-risk myelodysplastic syndrome (MDS) who are not responding to or cannot use ESA (a common anemia treatment). It may help improve anemia (low hemoglobin), reduce transfusions, and control the disease if you fit the specific criteria.
AmiensAges 18 years+ - NCT05444348Recruiting
Communication and care-planning support for blood cancer patients
This study tests an approach to help people with certain blood cancers and their main caregiver plan ahead and communicate more clearly about end-of-life care. It may help patients feel more supported and better prepared for difficult decisions.
AalborgAges 18 years+ - NCT05800210RecruitingPhase 2
T cell and B cell immune “wipeout” after stem cell transplant
This Phase 2 trial studies whether removing certain immune cells (alpha/beta T cells and CD19+ B cells) around the time of a stem cell transplant can improve outcomes for people with high-risk blood cancers. It may help by reducing the immune responses that can interfere with the transplant working, while keeping the process safe.
Gainesville, FloridaAges 6 months–39 years - NCT05924100RecruitingPhase 2
Luspatercept for lower red blood counts in MDS with del5q
This Phase 2 trial studies luspatercept to improve anemia (low red blood cells) in people with MDS that has a del5q change. It also checks safety, especially for people who need red blood cell transfusions and have not benefited from prior standard treatments.
AlessandriaAges 18 years+ - NCT05986240RecruitingPhase 1
Danvatirsen with or without venetoclax for advanced MDS or AML
This trial tests a new drug called danvatirsen, first alone and then combined with venetoclax, for people with advanced myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) that has come back or not responded to treatment. It aims to see if this approach can help when other options have stopped working.
The Bronx, New YorkAges 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 - NCT06378437RecruitingPhase 1
GLB-001 for blood cancers that have returned or not responded
This trial is testing a new drug called GLB-001 for people with certain blood cancers or bone marrow disorders that have not gotten better with standard treatments. The goal is to see if the drug is safe and how well it works.
Hefei, AnhuiAges 18 years+ - NCT06984536RecruitingPhase 2
Lowered immune drug plus mini transplant method for preventing graft vs host disease
This trial tests a new combination of medicines to prevent graft-versus-host disease (GVHD) in patients with leukemia or myelodysplastic syndromes receiving a stem cell transplant from a half-matched donor. It aims to see if using less anti-thymocyte globulin (ATG) plus a simpler post-transplant cyclophosphamide (PTCy) regimen works as well as standard care with fewer side effects.
BeijingAges 12–65 - NCT07155226RecruitingPhase 1/Phase 2
Study of AZD3632 for advanced blood cancers with certain gene changes
This trial is testing a new drug, AZD3632, either alone or with other cancer treatments, for people with advanced blood cancers that have specific genetic changes (like KMT2Ar, NPM1m, or others that cause HOX over-expression). The goal is to see if it can help when standard treatments have stopped working.
Decatur, IllinoisAges 16 years+ - NCT07270978RecruitingPhase 1
CD33-targeting cells for leukemia with leftover cancer cells
This trial tests a new treatment using special immune cells that target a protein called CD33 on leukemia cells. It's for people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) who still have small amounts of cancer cells left after other treatments.
Charlottesville, VirginiaAges 18 years+
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Common questions
- Are there clinical trials for refractory myelodysplastic syndrome?
- Yes. Clin2 currently lists 106 recruiting refractory 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 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 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.