Stem cell transplant offers hope for rare immune disorder
NCT ID NCT01861106
First seen Nov 01, 2025 · Last updated May 18, 2026 · Updated 29 times
Summary
This study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 receive chemotherapy or radiation before the transplant to prepare their body. The goal is to see if the donor cells can rebuild healthy blood and immune cells, but lifelong monitoring and possible medications may still be needed.
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 MDS are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Conditions
Explore the condition pages connected to this study.