Gene therapy survivors monitored for decades in new study

NCT ID NCT04049084

ENROLLING_BY_INVITATION Knowledge-focused Sponsor: University of California, Los Angeles Source: ClinicalTrials.gov ↗

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

Summary

This study follows 70 people who previously received gene therapy for a rare immune disorder called ADA-SCID. Researchers want to see how well the treatment worked over time and check for any long-term side effects. No new treatment is given—just regular health checkups.

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Conditions

The condition(s) this trial relates to.

severe combined immunodeficiency 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 DEFICIENCY SEVERE COMBINED IMMUNODEFICIENCY (SCID)

Contacts and locations

Locations

Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center

Los Angeles, California, 90095, United States
UCL Great Ormond Street Institute of Child Health

London, WC1N 1EH, United Kingdom

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