INHERITED BONE MARROW FAILURE SYNDROME
Clinical trials for INHERITED BONE MARROW FAILURE SYNDROME explained in plain language.
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Could a cancer drug fix a bleeding disorder and prevent leukemia?
Disease control Recruiting nowThis early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROME
Phase: PHASE1 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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New drug combo aims to stop blood cancer relapse after transplant in kids
Disease control Recruiting nowThis 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…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROME
Phase: PHASE2 • Sponsor: Franziska Wachter • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Massive study aims to uncover cancer secrets in rare bone marrow diseases
Knowledge-focused Recruiting nowThis natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROME
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 UTC
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New study aims to catch rare cancers early in fanconi anemia patients
Knowledge-focused Recruiting nowThis study follows people with Fanconi anemia, a rare inherited condition that raises the risk of certain cancers, especially squamous cell carcinoma. Researchers will screen 200 participants aged 12 and older every year for up to 10 years using exams, blood tests, and imaging to…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROME
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:05 UTC
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New study uses DNA tests to catch blood cancers before they start
Knowledge-focused Recruiting nowThis study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROME
Phase: NA • Sponsor: Mayo Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:07 UTC