for people ages up to 30 months (full criteria)
at San Francisco, California and other locations
study started
completion around



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).

Official 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)


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.


MPS-IH (Hurler Syndrome), MPS-IH, Hurler, MPS-I, Mucopolysaccharidoses, Mucopolysaccharidosis type I, Hurler syndrome, Mucopolysaccharidosis IH, Gene Therapy, Transplantation, Autologous Lentiviral vector, Mucopolysaccharidosis I, Syndrome, Experimental: OTL-203, Active Comparator: Allo-HSCT, Allo-HSCT


You can join if…

Open to people ages up to 30 months

  1. 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
  2. Confirmed laboratory diagnosis of MPS-IH as demonstrated by biallelic mutation(s) in the gene coding for IDUA enzyme
  3. Final confirmation of MPS-IH diagnosis by a Diagnostic Review Committee (DRC)

You CAN'T join if...

  1. Previous allo-HSCT or gene therapy
  2. Current enrollment or past treatment in any other interventional study/trial using a novel investigational agent
  3. Positivity to serological testing for Human Immunodeficiency Virus (HIV)-1 or HIV-2, Human T Lymphotropic Virus (HTLV)-1 or HTLV-2, Hepatitis B Virus (HBV) core, Hepatitis C Virus (HCV), mycoplasma, active tuberculosis (TB) and not meeting the microbiology biological screening requirements for drug product (DP) manufacturing.
  4. Malignant neoplasia (except local skin cancer)
  5. Myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)
  6. History of uncontrolled seizures
  7. Subjects with an active infection not responsive to treatment, end-organ damage, or any other disease that contraindicates performance of any of the procedures detailed in the protocol, or medical conditions or extenuating circumstances that, in the opinion of the Investigator, might compromise the subject's well-being or safety, or the interpretability of the subject's clinical data.
  8. Subjects, who in the opinion of the Investigator, may not be able to comply with protocol requirements or cooperate fully with the study procedures and necessary long- term follow up


  • UCSF Benioff Children's Hospital not yet accepting patients
    San Francisco California 94143 United States
  • University of Minnesota, Pediatrics accepting new patients
    Minnesota Minnesota 55455 United States


accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
Orchard Therapeutics
Phase 3 research study
Study Type
Expecting 40 study participants
Last Updated