New drug could make stem cell transplants safer for rare immune disease
NCT ID NCT05907746
First seen Nov 01, 2025 · Last updated Jun 17, 2026 · Updated 35 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 13 people 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 achieve lasting donor cell engraftment and restore normal blood cell production.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GATA2 DEFICIENCY are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.