Monocytopenia with susceptibility to infections
MONDO:0013607Also known as: MonoMAC, immunodeficiency type 21, monocytopenia and mycobacterial infection syndrome, DCML, Dendritic cell, monocyte, B lymphocyte, and natural Killer lymphocyte deficiency, GATA2 deficiency, IMD21, combined immunodeficiency with susceptibility to mycobacterial, viral, and fungal infections
18 clinical trials for this condition and its sub-types.
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New drug could make stem cell transplants safer for rare immune disorder
Disease control OngoingThis phase II trial tests whether a new drug called Briquilimab can make stem cell transplants safer for people with GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of infections and blood cancers. The study involves 13 participants a…
Phase: PHASE2 • Sponsor: National Institutes of Health Clinical Center (CC) • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Double transplant breakthrough offers hope for rare immune disorder patients
Disease control ENROLLING_BY_INVITATIONThis study tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC