MDS

This study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…

This phase 2 trial tests a stem cell transplant using a milder chemo and radiation regimen for people with blood cancers like AML and MDS. The goal is to see if this approach improves survival at 100 days after transplant. Participants receive donor stem cells along with drugs to…

This study tests whether adding a precise, targeted form of radiation to the standard chemotherapy and stem cell transplant can kill more cancer cells in the bone marrow and reduce the chance of relapse. About 27 adults with acute leukemia or myelodysplastic syndrome (MDS) will r…

This study tests whether giving two drugs, decitabine and filgrastim, after a stem cell transplant can help prevent blood cancers like AML and MDS from coming back in children and young adults. About 37 participants will receive the drug combination for up to 6 cycles. The main g…

This study tests a cord blood transplant in children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The goal is to see if this approach lowers the risk of death from treatment one year afte…

This study tests a combination of two drugs—luspatercept and erythropoietin (EPO)—for people with a low-risk form of myelodysplastic syndrome (MDS) that does not involve ring sideroblasts. Participants must have already tried EPO alone without success or be unable to take it. The…

This early-phase trial tests a new type of immune therapy called CD70-CAR T cells in children and young adults up to age 21 with blood cancers that have come back or not responded to treatment. The therapy involves taking the patient's own immune cells, engineering them to recogn…

This early-phase trial is testing a new drug called CG009301 in adults with blood cancers like leukemia that have returned or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can help. About 45 participants will receive the drug by …

This early-stage study tests an oral combination of tetrahydrouridine and decitabine in 20 adults with myelodysplastic syndromes (MDS) that returned or didn't respond to prior therapy. The main goals are to check safety and see if the drug improves blood cell counts and reduces h…

This early-stage trial tests a new treatment called MT-401-OTS for people with relapsed acute myeloid leukemia or high-risk myelodysplastic syndromes. The therapy uses donor immune cells specially trained to attack cancer, and is given as a one-time infusion. The main goals are t…

This early-phase study tests a new drug called eganelisib, given alone or with standard chemotherapy (cytarabine), in adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) that have returned or not responded to prior treatments. The main goals are …

This early-phase trial tests a new cell therapy called BSB-1001 in 38 adults with high-risk blood cancers (AML, ALL, or MDS) who are getting a stem cell transplant. BSB-1001 is made from donor immune cells that are modified to attack a specific marker on cancer cells. The goal is…

This study is testing a new blood test called PERIBLOOD-MDS that could diagnose a bone marrow disorder called MDS without needing a painful bone marrow biopsy. Researchers will compare results from the blood test with standard bone marrow exams in 1,500 adults with low blood coun…

This study looks at whether using a simple quality-of-life questionnaire to decide when to give blood transfusions can reduce the number of transfusions needed for people with advanced myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who are receiving palliative ca…

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study follows 1,000 people who previously received an experimental cell therapy (TSC-100 or TSC-101) for blood cancers like leukemia. Researchers will track side effects and survival for up to 15 years to see if the treatment remains safe and effective over time. No new trea…