Constitutional neutropenia
MONDO:0015134A rare disorder characterized by recurrent infantile infections and absence of neutrophils in the peripheral blood.
Also known as: congenital neutropenia, genetic infantile agranulocytosis, infantile genetic agranulocytosis, Kostmann disease, Kostmann neutropenia, Kostmann syndrome
65 clinical trials for this condition and its sub-types.
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Broader categories
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New shot aims to help kids with rare heart-muscle disease move easier
Disease control Not yet recruitingThis study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…
Phase: PHASE4 • Sponsor: Stealth BioTherapeutics Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:29 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
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New registry aims to unlock secrets of rare blood cancers
Knowledge-focused Not yet recruitingThis study is a registry that will collect medical information and blood samples from 500 adults with myelodysplastic syndromes (MDS) and related conditions. The goal is to learn more about how these diseases develop and progress over time. Participants will not receive any exper…
Sponsor: Technische Universität Dresden • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:28 UTC