New drug could make stem cell transplants safer for rare immune disorder
NCT ID NCT05907746
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 29 times
Summary
This study tests a new drug called Briquilimab to see if it can make stem cell transplants safer for people with GATA2 deficiency, a genetic condition that weakens the immune system and raises the risk of infections and blood cancers. About 7 participants aged 6 to 70 will receive Briquilimab before a donor stem cell transplant to help their body accept the new cells. The goal is to cure the disease with fewer side effects.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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