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A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects With Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care With Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)
Conditions
Interventions
Experimental: OTL-203
Active Comparator: Allo-HSCT
Locations
5
United States
University of Minnesota, Pediatrics
Minneapolis, Minnesota, United States
Ospedale San Raffaele
Milan, Italy
Princess Maxima Center
Utrecht, Netherlands
UMC Utrecht
Utrecht, Netherlands
Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital
Manchester, United Kingdom
Start Date
December 11, 2023
Primary Completion Date
March 1, 2028
Completion Date
March 1, 2031
Last Updated
December 22, 2025
Lead Sponsor
Orchard Therapeutics
Data Source & Attribution
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