New gene therapy aims to fix 'Bubble Boy' disease with a simple IV
NCT ID NCT03645460
Summary
This trial is testing a new gene therapy for children with a rare and life-threatening immune system disorder called ADA-SCID. Doctors will give patients a one-time intravenous (IV) infusion containing a modified virus that carries a corrected copy of the faulty gene. The goal is to see if this treatment is safe and can help the body rebuild its own immune system to fight off infections.
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 ADENOSINE DEAMINASE SEVERE COMBINED IMMUNODEFICIENCY (ADA-SCID) 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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Guilin Hospital of Chinese Traditional and Western Medicine
RECRUITINGGuilin, Guangxi, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.