One-Time injection aims to cure 'Bubble Boy' disease
NCT ID NCT03645460
Summary
This trial is testing a new gene therapy for children with a rare, life-threatening genetic disorder called ADA-SCID, which destroys the immune system. The goal is to deliver a working copy of the defective gene directly into the patient's bloodstream via a one-time intravenous injection, aiming to permanently correct the immune deficiency. Researchers will closely monitor 10 participants for safety and to see if their immune systems recover, allowing them to fight off infections.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Guilin Hospital of Chinese Traditional and Western Medicine
RECRUITINGGuilin, Guangxi, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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