Gene fix for rare immune disease shows promise in early trial
NCT ID NCT07113743
First seen Nov 01, 2025 · Last updated May 19, 2026 · Updated 26 times
Summary
This study tests a gene therapy for people with X-linked chronic granulomatous disease (X-CGD), a rare immune disorder that leads to severe infections. The treatment takes a patient's own stem cells, adds a working copy of the faulty gene, and returns them to the body after mild chemotherapy. Up to 10 participants aged 3–60 will be followed for safety and immune improvement over 2 years, with long-term monitoring for 13 more years.
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 CHRONIC GRANULOMATOUS DISEASE (CGD) 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
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
Explore the condition pages connected to this study.