Hemoglobin H disease
MONDO:0013512Alpha thalassemia caused by variation in three of the four copies of the alpha hemoglobin genes (e.g., large deletion in HBA1 and HBA2 genes in trans with a variant in either HBA1 or HBA2).
Also known as: Alpha-thalassemia intermedia, HBA1;HBA2 digenic triallelic hemoglobin H disease, HBH, HbH, HbH disease, alpha-thalassemia intermedia, haemoglobin H disease, deletional and nondeletional, hemoglobin H disease
51 clinical trials for this condition and its sub-types.
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New hope for kids: drug may prevent transplant complications
Disease control Not yet recruitingThis 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…
Phase: PHASE2 • Sponsor: Sung Won Choi • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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New pill could help kids with thalassemia fight anemia without transfusions
Disease control Not yet recruitingThis Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mi…
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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New CAR-T therapy targets antibodies blocking Life-Saving transplants
Disease control Not yet recruitingThis early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…
Phase: PHASE1, PHASE2 • Sponsor: Chang Yingjun • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Could a feeding tube after transplant save the gut?
Disease control Not yet recruitingThis study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …
Phase: PHASE2 • Sponsor: University of Nebraska • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC