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NCT06149403 · ClinicalTrials.gov registry record · Phase 3
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
A Phase 3 study of MPS-IH (Hurler Syndrome), sponsored by Orchard Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 41
- Enrollment target
- 5
- Study locations
NCT06149403: Active Phase 3 study of MPS-IH (Hurler Syndrome), sponsored by Orchard Therapeutics.
NCT06149403 is a Phase 3 study of MPS-IH (Hurler Syndrome) that is active but no longer recruiting, run by Orchard Therapeutics. The registered enrollment target is 41 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower). The trial reports 5 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06149403, a Phase 3 study of MPS-IH (Hurler Syndrome), is active but no longer recruiting, sponsored by Orchard Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 41 participants
- Enrollment target
- 5
- Study locations
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).
Primary Outcome
Defined by events of death, rescue transplant, treatment failure, immunological complications, severe cognitive and/or growth impairment.
Conditions Studied
Interventions
- GENETIC Experimental: OTL-203
- GENETIC Active Comparator: Allo-HSCT
Study Locations (5)
Other
- Ospedale San Raffaele - Milan
- Princess Maxima Center - Utrecht
- UMC Utrecht - Utrecht
- Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital - Manchester
Minnesota
- University of Minnesota, Pediatrics - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 41 participants |
| Start Date | 2023-12-11 |
| Est. Completion | 2031-03 |
| Phase | Phase 3 |
What the registry record for NCT06149403 still lists
NCT06149403 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 41 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower).
The record links to 1 condition, with MPS-IH (Hurler Syndrome) appearing as the primary indexed condition, and to 2 interventions - of which Experimental: OTL-203 is the first listed.
NCT06149403 lists 5 locations in 2 states (Other, Minnesota).
Frequently Asked Questions
What is clinical trial NCT06149403 about?
NCT06149403 is a clinical study titled "A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT". 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).
What is the current status of trial NCT06149403?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 41 participants. The study started on 2023-12-11. Estimated completion is 2031-03.
What conditions does trial NCT06149403 study?
This clinical trial studies the following conditions: MPS-IH (Hurler Syndrome).
What interventions are being tested in trial NCT06149403?
The interventions under investigation include: Experimental: OTL-203 (GENETIC), Active Comparator: Allo-HSCT (GENETIC).
Who is sponsoring clinical trial NCT06149403?
This trial is sponsored by Orchard Therapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06149403 being conducted?
This trial has 5 study locations across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT06149403's enrollment target sits among peer trials
41 1877th of 2000 higher than 123 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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