Gene therapy offers new hope for babies with rare 'Bubble Boy' disease

NCT ID NCT03538899

Recruiting now Disease control Sponsor: University of California, San Francisco Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a gene therapy for Artemis-deficient severe combined immunodeficiency (ART-SCID), a life-threatening immune disorder. It involves taking the patient's own blood stem cells, adding a working copy of the faulty gene, and infusing them back after mild chemotherapy. The goal is to rebuild a functioning immune system and improve survival. The trial enrolls 24 newly diagnosed infants and follows them for 15 years.

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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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Conditions

The condition(s) this trial relates to.

severe combined immunodeficiency severe combined immunodeficiency due to DCLRE1C deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SEVERE COMBINED IMMUNODEFICIENCY

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

University of California, San Francisco (UCSF) Children's Hospital
RECRUITING

San Francisco, California, 94143, United States