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Bubble boy disease gene therapy shows promise in new trial

NCT ID NCT05432310

Recruiting now Disease control Sponsor: University of California, Los Angeles Source: ClinicalTrials.gov ↗

First seen Apr 10, 2026 · Last updated May 23, 2026 · Updated 7 times

Summary

This study tests a gene therapy for children with ADA-SCID, a severe immune disorder often called 'bubble boy disease.' The treatment uses the child's own blood stem cells, which are modified in a lab to correct the genetic defect and then returned to the body. The goal is to restore immune function and improve survival. Up to 20 infants and children will take part.

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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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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • University of California, Los Angeles (UCLA)

    RECRUITING

    Los Angeles, California, 90095, United States

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

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

Conditions

Explore the condition pages connected to this study.

ADENOSINE DEAMINASE SEVERE COMBINED IMMUNE DEFICIENCY