MT2023-31: 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)


This research study is designed to compare a new gene therapy, known as OTL-203 (study drug), with a standard treatment called “allogeneic hematopoietic stem cell transplant” (allo-HSCT), to find out which is better for the treatment of MPS-IH.

I'm interested

Male or Female
Up to 18 years old
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• at least 28 days old to no more than 30 months old
• confirmed laboratory diagnosis of MPS-IH
• evidence of altered GAG metabolism
• see link to for complete inclusion and exclusion criteria
Exclusion Criteria:

• previous allo-HSCT or gene therapy
• diagnosis of HIV, Hepatitis B, Hepatitis C, or Mycoplasma
• history of uncontrolled seizures
• contraindications for MRI scans
• study staff will review additional exclusion criteria

Genetic: Active Comparator: Allo-HSCT, Genetic: Experimental: OTL-203

Rare Diseases

Hurler syndrome, MPS-IH, mucopolysaccharidosis type I

Ashish Gupta -
Ashish Gupta
See this study on