A Phase II, Open-Label, Study of Subcutaneous Canakinumab, an Anti-IL-1β Human Monoclonal Antibody, for Patients With Low or Int-1 Risk IPSS/IPSS-R Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia
ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Tagraxofusp and Azacitidine for Maintenance Treatment in Patients With CD123 Positive AML and MDS Following Donor Hematopoietic Cell Transplant
Study of MGUS, Smoldering Myeloma, Early MDS and CLL to Assess Molecular Events of Progression and Clinical Outcome
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Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Dose-Expansion Study of Low Dose Post-Transplant Cyclophosphamide/Tacrolimus/Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis in Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation
Lisaftoclax (APG-2575) Combined With Azacytidine (AZA) in the Treatment of Patients With Higher-risk Myelodysplastic Syndrome (GLORA-4).
Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
What is MDS?
Myelodysplastic syndromes, or MDS, are a group of bone marrow cancers in which blood stem cells are damaged and produce too few healthy red cells, white cells, or platelets. The result is anemia, infections, or bleeding, and many people need regular transfusions. MDS is uncommon before 50 and most often diagnosed in the 70s; prior chemotherapy or radiation and some chemical exposures raise the risk, but most cases have no known cause. Doctors classify it by risk using blood counts, the share of immature cells, and genetic changes. For lower risk disease the goal is to relieve anemia and reduce transfusions; for higher risk disease, to slow progression to acute myeloid leukemia with drugs such as azacitidine, and for fit patients a stem cell transplant, the only treatment that can cure it.
Types of MDS clinical trials
Anemia and transfusion trials. New drugs for lower risk MDS that raise red cell counts and reduce transfusions.
Higher risk MDS trials. Combinations with hypomethylating drugs, targeted drugs for specific mutations, and oral versions of standard treatment.
Precision medicine screening. National programs that test MDS genetics and assign people to matching trials.
Transplant trials. Conditioning regimens and relapse prevention; see stem cell therapy clinical trials.
Supportive care. Iron overload, infections, and quality of life during long term treatment.
MDS can progress to acute myeloid leukemia, and anemia from other causes is on our anemia page.
New to research? Read how clinical trials for cancer work.
Who can participate in MDS clinical trials?
Trials sort by risk group and treatment history. Lower risk trials usually enroll people with anemia who need transfusions or have stopped responding to standard drugs; higher risk trials often enroll newly diagnosed people before hypomethylating therapy or after it has stopped working. Many require bone marrow results including genetic testing, and some are designed for people 75 and older. Eligibility always varies by study.
Risk groups in MDS, and why they matter for trials
Very low and low risk. Untreated median survival in the original IPSS-R data about 8.8 and 5.3 years; trials focus on anemia and transfusions.
Intermediate risk. Median about 3 years; treated as lower or higher risk depending on other factors.
High and very high risk. Median about 1.6 and 0.8 years untreated; trials focus on slowing progression to AML and on transplant.
How to enroll in a MDS clinical trial
Identify your trial. Use the filters and match the stage or type and the treatment history named in study titles to your own.
Select your preferred location. Enter your city or state; large trials run at many sites, early phase studies at fewer centers.
Explore study details. Click "Learn More" for eligibility, prior treatment rules, and the visit schedule.
Complete the health profile. Click "Get started" to begin the 5-step application. Have three things ready: your risk score and bone marrow genetic results, your transfusion needs, and the treatments you have had.
Submit the application. A clinical trial coordinator reviews it and contacts you. Nothing is decided until informed consent, and participation is voluntary at every step.