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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

NCT ID: NCT06149403

Last Updated: 2025-12-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-12-11

Study Completion Date

2031-03-31

Brief Summary

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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

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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.

Conditions

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MPS-IH (Hurler Syndrome)

Keywords

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MPS-IH Hurler MPS-I Mucopolysaccharidoses Mucopolysaccharidosis type I Hurler syndrome Mucopolysaccharidosis IH Gene Therapy Transplantation Autologous Lentiviral vector

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Parallel assignment
Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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OTL-203

Eligible subjects randomized to Arm 1 will receive an intravenous (IV) infusion of OTL-203 gene therapy. Subjects will receive conditioning regimen with busulfan and fludarabine prior to OTL-203 infusion.

Group Type EXPERIMENTAL

Experimental: OTL-203

Intervention Type GENETIC

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

Allo-HSCT

Eligible subjects randomized to Arm 2 will receive allogeneic hematopoietic stem cell transplantation. Subjects will receive conditioning regimen with busulfan and fludarabine prior to allo-HSCT.

Group Type ACTIVE_COMPARATOR

Active Comparator: Allo-HSCT

Intervention Type GENETIC

Active Comparator: Allogeneic hematopoietic stem cell transplantation

Interventions

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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

Intervention Type GENETIC

Active Comparator: Allo-HSCT

Active Comparator: Allogeneic hematopoietic stem cell transplantation

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

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).

Exclusion Criteria

1. Previous allo-HSCT or gene therapy
2. 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
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.
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
Minimum Eligible Age

28 Days

Maximum Eligible Age

30 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Orchard Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Minnesota, Pediatrics

Minneapolis, Minnesota, United States

Site Status

Ospedale San Raffaele

Milan, , Italy

Site Status

Princess Maxima Center

Utrecht, , Netherlands

Site Status

UMC Utrecht

Utrecht, , Netherlands

Site Status

Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

Countries

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United States Italy Netherlands United Kingdom

Other Identifiers

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OTL-203-02

Identifier Type: -

Identifier Source: org_study_id