Syndromic constitutional thrombocytopenia
MONDO:0018795Also known as: syndromic constitutional thrombocytopenia
43 clinical trials for this condition and its sub-types.
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New drug UX016 targets rare muscle disease
Disease control Not yet recruitingThis early-stage trial tests UX016, a drug designed to help people with GNE myopathy, a rare genetic muscle-weakening disease. Researchers will give the drug or a placebo to 24 adults to see if it is safe and improves muscle strength. The study is not yet recruiting.
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:04 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