Status:
ACTIVE_NOT_RECRUITING
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
Lead Sponsor:
Orchard Therapeutics
Conditions:
MPS-IH (Hurler Syndrome)
Eligibility:
All Genders
28-30 years
Phase:
PHASE3
Brief 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...
Eligibility Criteria
Inclusion
- 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).
Exclusion
- Previous allo-HSCT or gene therapy
- Current enrollment or past treatment in any other interventional study/trial using a novel investigational agent and/or treated with prohibited medications listed in the protocol
- 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.
- Malignant neoplasia (except local skin cancer).
- Myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)
- History of uncontrolled seizures
- 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.
- 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
Key Trial Info
Start Date :
December 11 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2031
Estimated Enrollment :
41 Patients enrolled
Trial Details
Trial ID
NCT06149403
Start Date
December 11 2023
End Date
March 1 2031
Last Update
December 22 2025
Active Locations (5)
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1
University of Minnesota, Pediatrics
Minneapolis, Minnesota, United States, 55455
2
Ospedale San Raffaele
Milan, Italy, 20131
3
Princess Maxima Center
Utrecht, Netherlands, 3584 CS
4
UMC Utrecht
Utrecht, Netherlands, 3584 CX