![UMN logo](/assets/logo-14ca9c5bc764343ea0b1ec3edef3d39b15c438f8eb603b1a5fbf826128a74246.png)
StudyFinder
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)
![](https://studyfinder.umn.edu/rails/active_storage/blobs/redirect/eyJfcmFpbHMiOnsibWVzc2FnZSI6IkJBaHBBamdEIiwiZXhwIjpudWxsLCJwdXIiOiJibG9iX2lkIn19--449399a250764de5ca1b72aca520b5333c7a1499/Image%20107.jpg)
Recruiting
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.
Male or Female
Up to 18 years old
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 clinicaltrials.gov 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
Rare Diseases
Hurler syndrome, MPS-IH, mucopolysaccharidosis type I