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Bubble boy disease: gene therapy trial offers hope for immune system repair

NCT ID NCT05432310

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

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

Summary

This trial tests a gene therapy for children with ADA-SCID, a severe immune disorder often called 'bubble boy disease.' Doctors take the child's own blood stem cells, add a working gene, and return them via infusion. The goal is to restore immune function and improve survival. The study is currently recruiting 20 infants and children.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
gene therapy (EFS-ADA lentiviral vector modified stem cells)
What this could lead to
If successful, this could provide a one-time treatment that restores immune function in children with ADA-SCID, reducing the need for lifelong medication.
What could go wrong
This is an early-phase trial with only 20 participants, so results may not apply to all patients. Risks include side effects from the conditioning chemotherapy and the possibility that the gene therapy may not fully restore immunity.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency

As listed by the trial registrant

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

ADENOSINE DEAMINASE SEVERE COMBINED IMMUNE DEFICIENCY

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: •••••@•••••