# RGX-111 Gene Therapy in Patients With MPS I

> **NCT03580083** · PHASE1,PHASE2 · SUSPENDED · sponsor: **REGENXBIO Inc.** · enrollment: 21 (estimated)

## Conditions studied

- Mucopolysaccharidosis Type I (MPS I)
- Hurler Syndrome
- Hurler-Scheie Syndrome

## Interventions

- **GENETIC:** RGX-111

## Key facts

- **NCT ID:** NCT03580083
- **Lead sponsor:** REGENXBIO Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** SUSPENDED
- **Start date:** 2019-04-03
- **Primary completion:** 2027-07-07
- **Final completion:** 2029-03-06
- **Target enrollment:** 21 (ESTIMATED)
- **Why stopped:** The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.
- **Last updated:** 2026-03-12


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03580083

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03580083, "RGX-111 Gene Therapy in Patients With MPS I". Retrieved via AI Analytics 2026-05-29 from https://api.ai-analytics.org/clinical/NCT03580083. Licensed CC0.

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