Platelet-type bleeding disorder 15
MONDO:0014078Any inherited bleeding disorder, platelet-type in which the cause of the disease is a mutation in the ACTN1 gene.
Also known as: ACTN1 inherited bleeding disorder, platelet-type, BDPLT15, inherited bleeding disorder, platelet-type caused by mutation in ACTN1, platelet-type bleeding disorder 15, bleeding disorder, platelet-type, 15, macrothrombocytopenia, autosomal dominant, ACTN1-related
46 clinical trials for this condition and its sub-types.
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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