Gene therapy registry tracks lifelong safety for rare immune disorder

NCT ID NCT03478670

First seen May 19, 2026 · Last updated May 20, 2026 · Updated 1 time

Summary

This study follows 50 people with ADA-SCID, a severe immune disorder, who received Strimvelis gene therapy. Researchers will monitor participants for years to check for side effects, immune function, and overall health. The goal is to understand the long-term safety and how well the therapy works.

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Contacts and locations

Locations

  • Ospedale San Raffaele

    Milan, Lombardy, 20132, Italy

Conditions

Explore the condition pages connected to this study.