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Gene therapy could replace risky transplants for kids with hurler syndrome

NCT ID NCT06149403

Disease control Sponsor: Orchard Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 3 trial tests a new gene therapy called OTL-203 against the standard stem cell transplant for children with Hurler syndrome, a rare genetic disorder. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to improve survival and reduce complications. 41 participants are enrolled, and the study compares event-free survival between the two treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
OTL-203 (gene therapy using a patient's own stem cells modified to produce the missing IDUA enzyme)
What this could lead to
If successful, OTL-203 could become a safer and more effective alternative to standard stem cell transplant for children with Hurler syndrome, potentially reducing complications and improving outcomes.
What could go wrong
This is an early Phase 3 trial with only 41 participants, so results may not apply to all patients. Gene therapy carries risks like immune reactions or the treatment not working as expected.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

41 people

The number who actually took part.

Started

Dec 2023

Expected to finish

Mar 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

28 days to 30 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Hurler syndrome mucopolysaccharidosis Mucopolysaccharidosis I

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MPS-ih (hurler syndrome)

Contacts and locations

Locations

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

    Manchester, M13 9WL, United Kingdom

  • Ospedale San Raffaele

    Milan, 20131, Italy

  • Princess Maxima Center

    Utrecht, 3584 CS, Netherlands

  • UMC Utrecht

    Utrecht, 3584 CX, Netherlands

  • University of Minnesota, Pediatrics

    Minneapolis, Minnesota, 55455, United States