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)
Status: Active_not_recruiting
Location: See all (5) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 28 days
Maximum Age: 2
Healthy Volunteers: f
View:
• Norm-referenced cognitive standard score of ≥70 measured by age-appropriate cognitive domains of either Bayley Scale of Infant Development (BSID)-III or Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-IV.
• Confirmed laboratory diagnosis of MPS-IH as demonstrated by biallelic mutation(s) in the gene coding for IDUA enzyme
• Final confirmation of MPS-IH diagnosis by a Diagnostic Review Committee (DRC).
Locations
United States
Minnesota
University of Minnesota, Pediatrics
Minneapolis
Other Locations
Italy
Ospedale San Raffaele
Milan
Netherlands
Princess Maxima Center
Utrecht
UMC Utrecht
Utrecht
United Kingdom
Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital
Manchester
Time Frame
Start Date: 2023-12-11
Completion Date: 2031-03
Participants
Target number of participants: 41
Treatments
Experimental: OTL-203
Eligible subjects randomized to Arm 1 will receive an intravenous (IV) infusion of OTL-203 gene therapy. Subjects will receive conditioning regimen with busulfan and fludarabine prior to OTL-203 infusion.
Active_comparator: Allo-HSCT
Eligible subjects randomized to Arm 2 will receive allogeneic hematopoietic stem cell transplantation. Subjects will receive conditioning regimen with busulfan and fludarabine prior to allo-HSCT.
Related Therapeutic Areas
Sponsors
Leads: Orchard Therapeutics