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 108 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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+ - 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+ - 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 - NCT05986240RecruitingPhase 1
Danvatirsen plus venetoclax for advanced blood cancers
This trial tests a new drug called danvatirsen, first alone and then with venetoclax, for people with advanced acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have not responded to standard treatments. The goal is to see if this combination can help shrink or control the cancer.
The Bronx, New YorkAges 18 years+ - NCT07723703RecruitingPhase 1
Study of a new drug for acute myeloid leukemia and related blood cancer
This trial is testing a new medicine called AMX-883 for people with a type of blood cancer called acute myeloid leukemia (AML) or a high-risk blood disorder called myelodysplastic syndrome (MDS) that has come back or not responded to other treatments. The study hopes to see if the drug is safe and works, either alone or combined with another drug.
Fairfax, VirginiaAges 18 years+ - NCT07761533RecruitingPhase 1/Phase 2
PBSS1113 plus azacitidine for blood cancers
This study tests a new drug (PBSS1113) combined with azacitidine for people with certain blood cancers (AML or high-risk MDS). The goal is to see if the combination is safe and effective.
Hangzhou, ZhejiangAges 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+ - 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+ - NCT07588360Recruiting
Stem cell transplant for acute myeloid leukemia or myelodysplastic syndrome
This trial tests a type of stem cell transplant (using cells from a partially matched donor and umbilical cord blood) for patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The goal is to help patients whose disease is high-risk, has returned after treatment, or hasn't responded well to chemotherapy.
Fuzhou, FujianAges 14–60 - NCT07566377RecruitingPhase 2
Cord Blood Transplant for Children and Young Adults With Blood Cancer
This study tests whether using cord blood stem cells (collected from umbilical cords after birth) can help treat high-risk blood cancers in children and young adults when a matched donor is not quickly available. Cord blood transplants may help prevent cancer from coming back.
New York, New YorkAges Up to 26 years - NCT07565220RecruitingPhase 1
Thiotepa-based stem cell transplant for blood cancers
This trial tests a new chemotherapy preparation (using thiotepa) before a stem cell transplant from a matched donor to treat blood cancers like leukemia and myelodysplastic syndrome. The goal is to improve survival while reducing severe side effects.
Pittsburgh, PennsylvaniaAges 18 years+ - NCT07302776RecruitingPhase 1
Stopping immune suppression after stem cell transplant
This trial tests whether it's safe to stop one type of anti-rejection medication (tacrolimus) earlier than usual after a stem cell transplant from a matched donor. If you qualify, you may be able to reduce your long-term medication burden.
Palo Alto, CaliforniaAges 18–80 - 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+ - 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+ - 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 - 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+ - 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 - NCT02143830RecruitingPhase 2
Stem cell transplant chemotherapy for Fanconi anemia
This Phase 2 study tests a stem cell transplant approach using adjusted chemotherapy doses for people with Fanconi anemia. It may help replace damaged bone marrow and treat blood problems like severe aplastic anemia, MDS, or leukemia.
New York, New YorkAges 3 months+ - 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 - 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 - NCT05641259RecruitingPhase 1
LP-108 with azacitidine for blood cancers
This trial tests a new drug called LP-108 combined with azacitidine for people with certain blood cancers like acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML). It aims to see if the combination is safe and helps shrink or control the cancer.
NanchangAges 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+ - 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+ - 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+
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Common questions
- Are there clinical trials for refractory myelodysplastic syndrome?
- Yes. Clin2 currently lists 108 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.