# 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** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Orchard Therapeutics** · enrollment: 41 (actual)

## Conditions studied

- MPS-IH (Hurler Syndrome)

## Interventions

- **GENETIC:** Experimental: OTL-203
- **GENETIC:** Active Comparator: Allo-HSCT

## Key facts

- **NCT ID:** NCT06149403
- **Lead sponsor:** Orchard Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2023-12-11
- **Primary completion:** 2028-03
- **Final completion:** 2031-03
- **Target enrollment:** 41 (ACTUAL)
- **Last updated:** 2025-12-22


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06149403

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06149403, "A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT". Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/clinical/NCT06149403. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*