MedTrace Logo

RGX-111 Gene Therapy in Patients With MPS I

NCT03580083 · Status: SUSPENDED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2026-03-12

No results posted yet for this study

Summary

RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.

Conditions

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

Interventions

GENETIC

RGX-111

Recombinant adeno-associated virus serotype 9 capsid containing α-L-iduronidase expression cassette

Sponsors & Collaborators

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-03
Primary Completion
2027-07-07
Completion
2029-03-06
FDA Drug
Yes

Countries

  • United States
  • Brazil

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03580083 on ClinicalTrials.gov