Bubble boy disease tackled with one-shot gene therapy
NCT ID NCT03217617
First seen Nov 01, 2025 · Last updated Jun 10, 2026 · Updated 32 times
Summary
This trial tests a gene therapy for X-linked severe combined immunodeficiency (SCID-X1), also known as bubble boy disease. It involves a single IV injection of a modified virus to correct the faulty gene. The study aims to restore immune function and reduce infections in 10 patients who lack a suitable bone marrow donor.
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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.
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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
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.