Gene therapy aims to stop severe infections in rare immune disorder

NCT ID NCT03837483

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This trial tests a gene therapy called OTL-103 for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The therapy uses the patient's own blood stem cells, modified in a lab to carry a working copy of the faulty gene, then given back as a one-time infusion. The study will check if this reduces severe infections and bleeding episodes compared to before treatment.

What this could mean

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

Active substance: OTL-103 (gene therapy using the patient's own blood stem cells modified to carry a working copy of the WAS gene)
What this could lead to: If successful, this could offer a one-time treatment that reduces severe infections and bleeding episodes in people with Wiskott-Aldrich syndrome, potentially improving long-term health.
What could go wrong: This is a small early-phase trial with only 10 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or the modified cells not lasting long-term.

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.

Wiskott-Aldrich syndrome

As listed by the trial registrant

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

WISKOTT-ALDRICH SYNDROME

Contacts and locations

Locations

Children's Healthcare of Atlanta, Inc

Atlanta, Georgia, 30329, United States
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Milan, 20132, Italy

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