Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
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Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
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.