Inherited bone marrow failure syndromes
Clinical trials for Inherited bone marrow failure syndromes explained in plain language.
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New hope for kids: drug may prevent transplant complications
Disease control Recruiting nowThis study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROMES
Phase 2 • Sponsor: Sung Won Choi • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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20-Year study to track rare bone marrow diseases
Knowledge-focused Recruiting nowThis study follows people with acquired or inherited bone marrow failure for up to 20 years to learn how the diseases and treatments affect the body over time. Participants, aged 2 and older, will have regular check-ups and tests every 1 to 3 years. The goal is to better understa…
Matched conditions: INHERITED BONE MARROW FAILURE SYNDROMES
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Sep 16, 2026 00:00 UTC