A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)
A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
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Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Pre-emptive Therapy With DEC-C to Improve Outcomes in MDS Patients With Measurable Residual Disease Post Allogeneic Hematopoietic Cell Transplant
Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)
211^At-BC8-B10 Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, or Mixed-Phenotype Acute Leukemia
Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)
CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
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.