Gene therapy for 'Bubble Boy' disease under Long-Term watch

NCT ID NCT03478670

ENROLLING_BY_INVITATION Disease control Sponsor: Fondazione Telethon Source: ClinicalTrials.gov ↗

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

Summary

This study follows about 50 people with a rare immune disorder called ADA-SCID who have received a gene therapy treatment called Strimvelis. The goal is to track their health for many years to see if the treatment remains safe and effective. Researchers will monitor for side effects, immune function, and overall survival.

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Conditions

The condition(s) this trial relates to.

immunodeficiency disease inborn error of immunity 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.

IMMUNOLOGIC DEFICIENCY SYNDROMES

Contacts and locations

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Locations

Ospedale San Raffaele

Milan, Lombardy, 20132, Italy

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