Gene therapy may offer safer option for rare childhood disease
NCT ID NCT06149403
First seen Jan 07, 2026 · Last updated May 12, 2026 · Updated 22 times
Summary
This study tests a new gene therapy called OTL-203 for children with Hurler syndrome, a rare genetic disorder that damages organs and the brain. It compares the gene therapy to the current standard treatment, a stem cell transplant. The goal is to see if OTL-203 can improve survival and reduce serious complications like death, need for a rescue transplant, or severe cognitive and growth problems.
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This is a summary of
the original study
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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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Contacts and locations
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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
Conditions
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