OTL-203-02 / HURCULES (MPS-IH)

Clinical study of a new gene therapy (OTL-203) compared to a standard treatment as a safe and effective alternative to treat mucopolysaccharidosis Type I, Hurler syndrome (MPS-IH)

Active, not recruiting

Who can enter

Children with confirmed laboratory diagnosis of MPS-IH
Age at enrolment: ≥ 28 days to ≤30 months

Goal

The goal of this study is to compare the current standard treatment allogeneic hematopoietic stem cell transplantation (allo-HSCT) with the new gene therapy drug OTL-203. It is hypothesized that OTL-203 may offer advantages over standard treatment in terms of survival, reduction of complications and symptoms.

Background

Allo HSCT has been used as a standard treatment for MPS-IH by doctors around the world for many years, but it is not considered an approved drug by the regulators of medical treatments.

Treatment with OTL-203 or allo-HSCT is expected to stop or delay the progression of MPS-IH disease. There is the possibility of an improvement in quality of life, fewer MPS-IH symptoms and MPS-IH related complications, and reduction or stopping of other medications for MPS-IH complications.

In this study, the child will be given one of the two treatments and followed for a minimum of five years (up to 15 years with OTL-203 treatment). During the research, the child will also undergo various examinations at different time-points, including (but not limited to) a physical examination, blood tests, heart tests, functional and mobility examinations, and scans of brain or spine. After two years, there will be an interim evaluation of whether OTL-203 treatment can be a similar or improved treatment option.

OTL-203 is a gene therapy drug that consists of an autologous (from your own body), CD34+ cell-enriched population of hematopoietic stem and progenitor cells (HSPCs). The stem cells are modified outside the body to contain and produce the IDUA gene once back in the body. A single dose is administered back to the child via intravenous (i.v.) infusion. Reduced activity of the IDUA gene is considered an important factor in Hurler disease.

Currently, an ongoing phase I/II study shows that OTL-203 is well tolerated, and follow-up results suggest an encouraging outcome.

This study is now closed for inclusion.

Last reviewed

March 23, 2026

Study details
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)
Short title
A study to investigate the efficacy and safety of OTL-203 in subjects with MPS-IH compared with standard of care with allogeneic HSCT
Condition
Mucopolysaccharidosis Type I, Hurler syndrome (MPS-IH)
Phase
3
Maximum number of patients
40, of whom 4 are expected to participate in the Netherlands
Start date
September 24, 2024
Status
Closed for inclusion
Local principal investigator
Dr. C. Lindemans
Sponsor
Orchard Therapeutics (Europe) Ltd, UK
Approval
The study of this new treatment has been reviewed by an accredited medical research ethics committee. This committee has decided that it is justified to ask patients to participate in this study. More information can be found at: CCMO.
Trial registry number
EU clinical trial register: 2022-500306-17-00
Clinicaltrials.gov: NCT06149403