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Active Not RecruitingPhase 3

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403

A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).

MPS-IH (Hurler Syndrome)

Orchard Therapeutics41 participantsStarted Dec 2023

Minneapolis, Minnesota, United States • Milan, Italy • Utrecht, Netherlands +2 more locations

Data from ClinicalTrials.govTerms