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Clinical trial

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)

NCT06149403 is registered in ClinicalTrials.gov with Orchard Therapeutics as lead sponsor.

NCT NCT06149403
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Registry record

As filed in ClinicalTrials.gov.

Title
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)
Lead sponsor
Orchard Therapeutics
Phase
Phase 3
Overall status
Active, not recruiting
Start date
December 11, 2023
Completion date
March 31, 2031
Enrollment
41 (actual)
Sites
5

Conditions

As the registry lists them; a condition with its own page is linked.

mps-ih (hurler syndrome)