# Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

> **NCT03488394** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **Orchard Therapeutics** · enrollment: 8 (actual)

## Conditions studied

- Mucopolysaccharidosis IH

## Interventions

- **GENETIC:** Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.

## Key facts

- **NCT ID:** NCT03488394
- **Lead sponsor:** Orchard Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2018-05-11
- **Primary completion:** 2028-01
- **Final completion:** 2028-03
- **Target enrollment:** 8 (ACTUAL)
- **Last updated:** 2025-12-09

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03488394

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03488394, "Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT03488394. Licensed CC0.

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