Gene therapy could replace risky transplants for kids with hurler syndrome
NCT ID NCT06149403
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 3 trial tests a new gene therapy called OTL-203 against the standard stem cell transplant for children with Hurler syndrome, a rare genetic disorder. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to improve survival and reduce complications. 41 participants are enrolled, and the study compares event-free survival between the two treatments.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- OTL-203 (gene therapy using a patient's own stem cells modified to produce the missing IDUA enzyme)
- What this could lead to
- If successful, OTL-203 could become a safer and more effective alternative to standard stem cell transplant for children with Hurler syndrome, potentially reducing complications and improving outcomes.
- What could go wrong
- This is an early Phase 3 trial with only 41 participants, so results may not apply to all patients. Gene therapy carries risks like immune reactions or the treatment not working as expected.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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Ospedale San Raffaele
Milan, 20131, Italy
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Princess Maxima Center
Utrecht, 3584 CS, Netherlands
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UMC Utrecht
Utrecht, 3584 CX, Netherlands
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University of Minnesota, Pediatrics
Minneapolis, Minnesota, 55455, United States