Condition
Myelodysplastic Syndrome clinical trials and research
As of November 12, 2026, Find a Trial lists 64 Myelodysplastic Syndrome clinical trials that are recruiting, at sites in 35 countries (most in United States (50), China (6), Canada (4)). By phase: Phase 2 (28), Phase 1 (20), Phase 1/2 (3), Phase 2/3 (2). The most active sponsors are M.D. Anderson Cancer Center (11), City of Hope Medical Center (9), Fred Hutchinson Cancer Center (5). There are also 11 NIH-funded research projects on Myelodysplastic Syndrome, with $4.6 million in listed awards. Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 64Clinical trials
- 11NIH research projects
- 35Countries
Newest Myelodysplastic Syndrome clinical trials
All recruiting trials (64)
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Low-dose Liposomal Amphotericin B for preventing fungal infections in adults with blood cancers and prolonged neutropenia
This study tests whether giving a daily 50 mg dose of liposomal amphotericin B can help prevent invasive fungal infections in adults with blood cancers who have prolonged low white blood cell counts.
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Total marrow and lymphoid irradiation with fludarabine and melphalan for older adults with refractory/relapsed AML or high-risk MDS undergoing donor stem cell transplant
This phase 2 trial tests a targeted radiation conditioning (TMLI) with fludarabine and melphalan before a matched donor stem cell transplant in people aged 50 or older with difficult-to-treat AML or high-risk MDS.
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Nicotinamide Riboside and Pterostilbene in lower risk MDS and high-risk CCUS
This phase 2 study tests whether nicotinamide riboside plus pterostilbene can help improve blood counts in adults with lower risk MDS or high-risk CCUS who are not transfusion-dependent.
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QUAIL-100 in kids and young adults after stem cell transplant with high-risk blood cancers
This study tests QUAIL-100 in pediatric and young adult patients who have high-risk acute leukemia or myelodysplastic syndrome and have already received a partially matched stem cell transplant.
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Elritercept versus epoetin alfa for transfusion-dependent anemia in lower-risk MDS
This study tests whether elritercept can reduce the need for red blood cell transfusions and improve fatigue compared with epoetin alfa in adults with very low-, low-, or intermediate-risk myelodysplastic syndromes who need regular transfusions.
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CD64 CAR T Therapy for Adults with Relapsed or Refractory AML
This phase 1 study tests a patient’s own CD64-directed CAR T cells to see if they can be safely given and potentially help adults with relapsed or refractory AML.
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In-home cancer treatment with remote specialist support for patients in the Florida Panhandle
This program will try giving standard cancer treatments at home to people living in the Florida Panhandle and nearby areas to see if they prefer it and have better treatment satisfaction.
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Adding FHD-286 to low‑dose decitabine and venetoclax for adverse‑risk AML
This trial tests whether adding FHD-286 to a low‑dose decitabine plus venetoclax regimen helps people with newly diagnosed adverse‑risk acute myeloid leukemia or AML that progressed after one prior therapy.
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Prophylactic DLI-X to Prevent Leukemia Relapse After Transplant
This first-in-human trial tests a prophylactic DLI-X approach to see if it can prevent relapse after matched sibling or haploidentical stem cell transplant in patients with acute leukemias and related blood disorders.
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Induction and consolidation using fludarabine, cytarabine, idarubicin, and venetoclax for acute myeloid leukemia
This trial tests whether adding venetoclax to induction and consolidation chemotherapy (fludarabine, cytarabine, and idarubicin) helps adults 18–65 with newly diagnosed acute myeloid leukemia or high-blast myelodysplastic syndrome achieve deeper remissions than standard cytarabine/daunorubicin therapy.
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High-altitude stem cell and bone marrow transplant registry on the Qinghai-Tibet Plateau
This project will collect clinical data and blood/bone marrow samples from people having hematopoietic stem cell transplants on the Qinghai‑Tibet Plateau to see how high altitude affects transplant outcomes and recovery.
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RPT1G for adults with relapsed or refractory acute myeloid leukemia and high-risk MDS
This study will test whether the drug RPT1G is safe and find the best dose for adults whose AML or high-risk MDS has come back or not responded to treatment.
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FLAG‑Ida chemotherapy followed by reduced‑intensity total‑body irradiation and donor stem cell transplant for adults 60+ with adverse‑risk AML or other high‑grade myeloid cancers
This trial tests whether FLAG‑Ida chemotherapy given immediately before lower‑intensity total‑body radiation and a donor stem cell transplant can improve outcomes for adults 60 and older with newly diagnosed adverse‑risk acute myeloid leukemia or similar high‑grade myeloid cancers.
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CMV Triplex vaccine to boost immunity and prevent CMV after haploidentical stem cell transplant
This trial tests whether giving a CMV-MVA Triplex vaccine to donors and/or recipients, with or without the antiviral letermovir, can boost CMV immunity and prevent CMV viremia in people having haploidentical stem cell transplants.
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Adding emapalumab to standard post-transplant medicines to prevent graft‑versus‑host disease after reduced‑intensity donor stem cell transplant
This trial will test whether adding emapalumab to post‑transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil can prevent graft‑versus‑host disease in adults with AML or MDS receiving a reduced‑intensity donor stem cell transplant.
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Lower-dose ATG plus mini post‑transplant cyclophosphamide to prevent GVHD after haploidentical stem cell transplant
This approach tries a lower dose of ATG combined with a short course of post‑transplant cyclophosphamide to prevent graft‑versus‑host disease in people aged 12–65 receiving a haploidentical stem cell transplant for acute leukemia or myelodysplastic syndrome.
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Evaluating XS-04 for patients with relapsed or refractory blood cancers
This study is testing a new tablet called XS-04 to see if it is safe and how well it works for people with certain types of blood cancers that have come back or didn’t respond to other treatments.
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Vedolizumab with post-transplant cyclophosphamide and short-course tacrolimus to prevent graft-versus-host disease after reduced-intensity allogeneic stem cell transplant
This trial tests whether adding vedolizumab to post-transplant cyclophosphamide and a short course of tacrolimus can prevent graft-versus-host disease in adults getting reduced-intensity allogeneic stem cell transplants for leukemias or related blood disorders.
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HPV vaccination after allogeneic stem cell transplant
This trial will test whether giving the 9‑valent HPV vaccine early or later after an allogeneic stem cell transplant produces a protective antibody response in adults up to age 45 who had a transplant.
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Extracorporeal iron purification with MEX-CD1 for patients with MDS or myelofibrosis
This study will try using an external blood-filter device (MEX-CD1) to remove excess iron in transfusion-dependent patients with myelodysplastic syndrome or myelofibrosis who cannot tolerate standard chelation.
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Exercise for patients with low-risk myeloid cancers
This study is testing if a special exercise program can help people with low-risk myeloid cancers stay healthy and possibly prevent their condition from getting worse.
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Preventing GVHD in children and young adults with blood cancers
This study is testing a new treatment plan to see if it can help prevent graft-versus-host disease in children and young adults with blood cancers after they receive a transplant.
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Testing a new treatment for patients with specific myeloid cancers
This study is testing a new treatment combining rezatapopt and azacitidine to see if it can help patients with specific myeloid cancers, like acute myeloid leukemia and myelodysplastic syndrome, feel better and live longer.
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Combining Enasidenib with Standard Treatment for Higher-Risk Myelodysplastic Syndrome
This study is testing if adding a new drug called enasidenib to standard treatment can help people with higher-risk myelodysplastic syndrome and an IDH2 mutation achieve better remission rates.
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Using olutasidenib to prevent relapse in patients with IDH1 mutated blood cancers after transplant
This study is testing if a new drug called olutasidenib can help prevent blood cancers from coming back in patients with a specific gene mutation after they have had a stem cell transplant.
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Evaluating risks of myelodysplastic syndrome in patients receiving peptide radionuclide therapy
This study is trying to find out if certain patients who receive a specific cancer treatment are at risk of developing blood disorders like myelodysplastic syndrome and leukemia, to make the treatment safer for everyone.
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Using tagraxofusp and azacitidine to prevent cancer recurrence after transplant in certain leukemia and syndrome patients
This study is testing if a new combination of two drugs can help prevent cancer from coming back in people with certain types of leukemia and blood disorders after they have had a stem cell transplant.
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Testing a new anti-cancer drug for patients with acute myeloid leukemia and myelodysplastic syndromes
This study is testing a new anti-cancer drug called cirtuvivint, alone and with another drug, to see if it helps people with acute myeloid leukemia and myelodysplastic syndromes feel better and live longer.
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Restoring gut bacteria before stem cell transplant for blood cancer patients
This study is testing if restoring gut bacteria with a special treatment before stem cell transplants can help blood cancer patients have better health and treatment outcomes.
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Evaluating a new treatment for recurrent adult acute myeloid leukemia and myelodysplastic syndrome
This study is testing a new combination treatment for adults with recurrent acute myeloid leukemia and myelodysplastic syndrome to see if it works better than current options.
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Using a new antibody treatment with chemotherapy and radiation for high-risk blood cancers before stem cell transplant
This study is testing a new antibody treatment combined with chemotherapy and radiation to see if it can help people with high-risk blood cancers before they receive a stem cell transplant.
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Preventing Acute GVHD After Stem Cell Transplant
This study is testing a new way to give a medication before and during stem cell transplants to see if it can help prevent a serious condition called acute GVHD in patients.
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Using ruxolitinib, tacrolimus, and methotrexate to prevent graft versus host disease in young patients after bone marrow transplant
This study is testing if a combination of three medications can help prevent graft versus host disease in young patients after they receive a bone marrow transplant for certain types of leukemia or blood disorders.
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Assessing the safety of luspatercept in Korean patients with myelodysplastic syndrome or beta thalassemia
This study is testing how safe the drug luspatercept is for adult Korean patients with myelodysplastic syndrome or beta thalassemia while they are being treated.
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Curcumin for treating inflammation and symptoms in blood disorders
This study is testing if a curcumin supplement can help reduce inflammation and improve symptoms in people with certain blood disorders.
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Vaccination to prevent CMV infection in stem cell transplant patients
This study is testing a new vaccine to see if it can help prevent CMV infections in patients receiving stem cell transplants from related donors.
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Using cord blood transplant and chemotherapy to treat high-risk blood cancers
This study is testing if combining umbilical cord blood transplants with certain chemotherapy treatments can help people with high-risk blood cancers do better and have fewer side effects.
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At-home cancer treatment versus clinic-based treatment for advanced cancer patients
This study tests whether giving cancer treatment at home is better for comfort and satisfaction compared to getting it at a clinic for people with advanced cancer.
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SP-420 to remove excess iron in adults with transfusion-dependent beta thalassemia or low‑risk MDS
This will test whether SP-420 can clear excess liver iron in adults who receive regular transfusions for beta thalassemia or transfusion‑dependent low‑risk myelodysplastic syndrome.
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Screening to match myeloid cancer patients with treatment options
This study is testing a new way to help people with acute myeloid leukemia or myelodysplastic syndrome find the right treatment options faster by checking their blood and bone marrow for specific markers.
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Reduced-intensity conditioning to lower treatment-related mortality in stem cell transplant patients
This study is testing a new way to prepare patients for a stem cell transplant to see if it can lower the risk of serious treatment-related complications.
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Combination treatment for FLT3-mutated acute myeloid leukemia and high-risk myelodysplastic syndrome
This study is testing a new combination of three drugs to see if it can help people with FLT3-mutated acute myeloid leukemia or high-risk myelodysplastic syndrome feel better and live longer.
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Screening for pediatric leukemia in children with relapsed or difficult-to-treat cases
This study is testing how to better screen children and young adults with tough cases of leukemia to see if they qualify for new treatment options.
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Using venetoclax with busulfan to treat high-risk blood cancers
This study is testing if combining venetoclax with busulfan, cladribine, and fludarabine can help people with high-risk blood cancers like acute myeloid leukemia and myelodysplastic syndrome do better after stem cell transplantation.
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Long-term study of blood diseases in patients in China
This study is trying to gather detailed information from patients in China with blood diseases like leukemia and multiple myeloma to better understand how these conditions affect their lives and treatment options.
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Combining CPX-351 and ivosidenib for treating IDH1 mutated acute myeloid leukemia and high-risk myelodysplastic syndrome
This study is testing a combination of two treatments, CPX-351 and ivosidenib, to see if they can help people with acute myeloid leukemia or high-risk myelodysplastic syndrome who have an IDH1 mutation.
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Combining decitabine with ruxolitinib, fedratinib, or pacritinib for treating advanced myeloproliferative neoplasms
This study is testing if combining decitabine with other medications can help patients with advanced blood cancers get ready for a stem cell transplant.
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Evaluating BTX-A51 for patients with relapsed or refractory acute myeloid leukemia or high-risk myelodysplastic syndrome
This study is testing a new drug called BTX-A51 to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia or high-risk myelodysplastic syndrome feel better and improve their treatment options.
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Treatment of low or intermediate-risk myelodysplastic syndromes and chronic myelomonocytic leukemia with canakinumab
This study is testing if a new medication called canakinumab can help people with low or intermediate-risk myelodysplastic syndromes and chronic myelomonocytic leukemia feel better and reduce their need for blood transfusions.
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Donor stem cell transplant with chemotherapy and radiation for blood cancers
This study is testing if a donor stem cell transplant combined with different doses of chemotherapy and radiation can help people with blood cancers recover better.
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Determining the best dose of CYAD-02 for patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome
This study is testing different doses of a new treatment called CYAD-02 to see which one works best for people with relapsed or hard-to-treat acute myeloid leukemia or myelodysplastic syndrome.
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Strategies to reduce graft versus host disease in blood stem cell transplant patients with acute leukemia or MDS
This study is testing two different treatment plans to see which one better helps prevent graft versus host disease in patients with acute leukemia or myelodysplastic syndrome after they receive a blood stem cell transplant.
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Using enasidenib and azacitidine to treat patients with recurrent or refractory acute myeloid leukemia and IDH2 gene mutation
This study is testing if a combination of two medications, enasidenib and azacitidine, can help people with recurrent or hard-to-treat acute myeloid leukemia that has an IDH2 gene mutation.
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Using edetate calcium disodium or succimer to treat acute myeloid leukemia or myelodysplastic syndrome during chemotherapy
This study is testing whether two drugs, edetate calcium disodium and succimer, can safely help people with acute myeloid leukemia or myelodysplastic syndrome feel better during chemotherapy.
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Combining Ivosidenib and Venetoclax with or without Azacitidine for treating IDH1 mutated blood cancers
This study is testing a combination of two drugs, ivosidenib and venetoclax, with or without azacitidine, to see if it helps people with certain types of blood cancers that have an IDH1 mutation.
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HA-1 TCR T cell therapy for leukemia that returned or didn't respond after a donor stem cell transplant
This Phase 1 study will test whether giving specially engineered donor T cells called HA-1 TCR T cells is safe and what dose works for people whose acute leukemia came back or didn't respond after a donor stem cell transplant.
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Combining SL-401 with Azacitidine or Venetoclax for treating blood cancers
This study is testing if combining a new treatment called SL-401 with existing drugs azacitidine or venetoclax can help people with certain blood cancers feel better and improve their outcomes.
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Vaccine treatment for relapsed or refractory blood cancers
This study is testing a new vaccine treatment for people with relapsed or hard-to-treat blood cancers to see if it can help them feel better and live longer.
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Personalized NK cell therapy after umbilical cord blood transplant for leukemia
This study is testing if a special treatment using personalized immune cells from umbilical cord blood can help people with leukemia feel better after their transplant.
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Transplantation with modified stem cells and Zoledronate for pediatric cancers
This study is testing a new way to use modified stem cells and a drug called Zoledronate to see if it can safely help children with tough-to-treat cancers.
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Combining cladribine, idarubicin, cytarabine, and venetoclax for treating certain blood cancers
This study is testing a new combination of four medications to see if it helps people with certain types of blood cancers feel better and live longer.
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Treating Aplastic Anemia and Myelodysplastic Syndrome with Combination Therapy
This study is testing a combination of four treatments to see if it can help people with aplastic anemia or certain types of myelodysplastic syndrome improve their blood counts and overall health.
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Monitoring minimal residual disease in patients with acute myeloid leukemia or myelodysplastic syndrome
This study is trying to see if new tests can help find hidden cancer cells in patients with acute myeloid leukemia or myelodysplastic syndrome who seem to be in remission, to better understand their risk of relapse.
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Collection of tissue samples for cancer research
This study is collecting tissue samples from adults and kids with cancer to help researchers learn more about the disease and improve treatments.
NIH-funded research (11)
Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.
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Targeting diverse stem cells in myelodysplastic syndrome to eradicate the disease
Aimed at identifying weaknesses in the diverse stem cell populations of myelodysplastic syndrome so future treatments can wipe out the root cause of the disease.
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New treatment approach for myelodysplastic syndrome and acute myeloid leukemia
This project aims to develop a new antibody-based therapy to help people with MDS or AML, especially older or frail patients who cannot tolerate high-dose chemotherapy.
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Targeting metabolic weak spots in myelodysplastic syndrome stem cells
The team is trying metabolism-based treatments to kill the stem cells that drive high-risk myelodysplastic syndrome (MDS).
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Investigating the role of PI3 kinase in Myelodysplastic Syndrome
This study is looking at how certain changes in genes might affect blood cell production in older adults with Myelodysplastic Syndrome (MDS), and it hopes to find new ways to help improve treatment for people with this condition.
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Identifying harmful and harmless genetic variations in blood cancer predisposition genes
This study is looking at how certain changes in a gene called DDX41 might affect the chances of developing blood disorders like myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), so that doctors can provide better advice and support for patients at risk.
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Investigating CCRL2's role in myelodysplastic syndrome
This study is looking at how a specific gene called CCRL2 affects the growth of harmful cells in myelodysplastic syndrome (MDS), especially in men, to find new ways to improve treatment for people with this blood disorder.
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Understanding how immune evasion occurs in high-risk myelodysplastic syndrome
This study is looking at how a protein called VISTA helps high-risk myelodysplastic syndrome (HR-MDS) avoid being attacked by the immune system, with the goal of finding new ways to improve treatments for patients like you.
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Targeting specific stem cells in myelodysplastic syndrome for better treatment outcomes
This study is looking to find better ways to treat high-risk myelodysplastic syndrome (MDS) by understanding the different types of stem cells involved in the disease and testing a new combination of drugs to help improve patient outcomes.
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New treatment options for myelodysplastic syndrome and acute myeloid leukemia
This study is exploring new treatment options for people with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) by looking at their genetic makeup and developing targeted therapies that could help improve their health.
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Understanding how U2AF1 mutations contribute to myelodysplastic syndrome
This study is looking into how changes in the U2AF1 gene affect people with myelodysplastic syndrome (MDS), which could help us find better ways to diagnose and treat this blood disorder.
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Targeting CD117 with Astatine-211 to treat Acute Myeloid Leukemia and Myelodysplastic Syndrome
This study is testing a new treatment that uses a special combination of antibodies and a radioactive substance to help patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) by targeting cancer cells more directly while protecting healthy ones.
Related topics
Quick answers
How many Myelodysplastic Syndrome clinical trials are recruiting right now?
64, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 35 countries. The most sites are in United States (50 trials), China (6 trials), Canada (4 trials), Puerto Rico (3 trials), Australia (2 trials). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 2: 28, Phase 1: 20, Phase 1/2: 3, Phase 2/3: 2. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.