Gene therapy aims to stop severe infections in rare immune disorder
NCT ID NCT03837483
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Healthcare of Atlanta, Inc
Atlanta, Georgia, 30329, United States
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Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, 20132, Italy