NCT06149403

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

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 41 (actual)
• Sponsor: Orchard Therapeutics · Industry
• Sex: All
• Age: 28 Days – 30 Months
• Healthy volunteers: Not accepted

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).

Detailed description

The study is a multi-center, randomized, active controlled clinical trial designed to evaluate the efficacy and safety of OTL-203 in patients with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT). A total of 40 patients with a confirmed diagnosis of MPS-IH who meet the study inclusion criteria will be randomized to receive either OTL-203 or allo-HSCT. The trial will comprise of a screening, baseline, and treatment period, with a follow-up period of 5 years post-treatment, and primary analysis performed at 2 years follow-up of the last treated subject.

Conditions

• MPS-IH (Hurler Syndrome)

Interventions

Type	Name	Description
GENETIC	Experimental: OTL-203	Experimental: OTL-203: Autologous CD34+ enriched cell fraction that contains hematopoietic stem and progenitor cells transduced ex vivo using lentiviral vector encoding the human IDUA gene
GENETIC	Active Comparator: Allo-HSCT	Active Comparator: Allogeneic hematopoietic stem cell transplantation

Timeline

• Start date: 2023-12-11
• Primary completion: 2028-03-01
• Completion: 2031-03-01
• First posted: 2023-11-29
• Last updated: 2025-12-22

Locations

5 sites across 4 countries: United States, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study