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

NCT06149403 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2025-12-22

No results posted yet for this study

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).

Conditions

  • MPS-IH (Hurler Syndrome)

Interventions

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

Sponsors & Collaborators

  • Orchard Therapeutics

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
PARALLEL

Eligibility

Min Age
28 Days
Max Age
30 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-12-11
Primary Completion
2028-03-31
Completion
2031-03-31
FDA Drug
Yes

Countries

  • United States
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06149403 on ClinicalTrials.gov