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
- NCT04256317RecruitingPhase 2/Phase 3
Azacitidine pills with or without venetoclax for certain blood cancers
This study tests whether a pill form of azacitidine (ASTX030) helps people with myelodysplastic syndromes (MDS), CMML, or AML. Some groups may also receive venetoclax; the goal is better blood cancer control for people who aren’t good candidates for intensive chemotherapy.
Los Angeles, CaliforniaAges 18 years+ - NCT03471260RecruitingPhase 1/Phase 2
Ivosidenib and venetoclax for IDH1-mutated leukemia or MDS
This Phase 1/2 study tests whether the medicines ivosidenib and venetoclax (with or without azacitidine) can help people with certain “IDH1-mutated” blood cancers like AML or high-risk MDS. It’s meant for patients whose disease has come back, doesn’t respond, or who can’t safely get standard chemotherapy.
Boston, MassachusettsAges 18 years+ - NCT03874052RecruitingPhase 1
Ruxolitinib plus venetoclax and azacitidine for relapsed AML
This early-phase study tests a combination of ruxolitinib with venetoclax, with or without azacitidine, for adults whose acute myeloid leukemia (AML) has come back or does not respond to treatment. It may help by stopping cancer cells from growing and improving blood counts, with the exact plan guided by the study arm you’re assigned to.
Columbus, OhioAges 18 years+ - NCT06820268RecruitingPhase 1
XS-04 for blood cancers that returned after treatment
This study tests a new drug, XS-04, for people with certain blood cancers (like B-cell lymphoma, AML, or MDS) that have come back or not responded to standard treatments. The goal is to see if the drug is safe and can help shrink tumors or control the disease.
BeijingAges 18–75 - NCT07347171RecruitingPhase 1
Testing CG009301 for blood cancers that returned or didn't respond
This trial tests a new drug called CG009301 for people with blood cancers that have come back or haven't responded to standard treatments. The goal is to see if the drug is safe and works against these cancers.
NanchangAges 18–75 - 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+ - 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+ - 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+ - NCT07285668Recruiting
Special donor immune cells after stem cell transplant for high-risk blood cancers
This study tests whether giving specially treated donor immune cells after a stem cell transplant can help prevent relapse in patients with high-risk blood cancers. You would get a stem cell transplant from a relative, followed by an infusion of donor cells that have been processed to reduce the risk of graft-versus-host disease.
Madison, WisconsinAges 18–80 - NCT06952803RecruitingPhase 3
Testing a new drug for men with BRCA-related prostate cancer
This trial tests whether adding a new drug (saruparib) to standard radiation and hormone therapy can help prevent prostate cancer from spreading to other parts of the body. It is for men with high-risk prostate cancer that has a BRCA gene mutation.
Phoenix, ArizonaAges 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 - 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 - 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+ - 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+ - 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+ - 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+ - 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 - 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 - NCT04771572RecruitingPhase 1
Oral drug LP-118 for relapsed blood cancers
This Phase 1 study tests an oral (by mouth) drug called LP-118 in people whose blood cancers have returned or did not respond to prior treatment. The goal is to find a safe dose and see whether it can shrink or control the cancer.
Chicago, IllinoisAges 13 years+ - NCT05890352RecruitingPhase 2
Add new medicines to standard treatment for returning lymphoma
This Phase 2 study tests whether adding tafasitamab, lenalidomide, tazemetostat, and zanubrutinib (along with standard lymphoma treatment) can improve outcomes in people whose large B-cell lymphoma has returned or not responded. It may help control disease more effectively and improve response duration for eligible patients.
Tucson, ArizonaAges 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.