NCT03580083View on ClinicalTrials.gov

RGX-111 Gene Therapy in Patients With MPS I

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

8

Participants

Timeline

Start Date

April 3, 2019

Primary Completion Date

March 1, 2023

Study Completion Date

October 31, 2024

Conditions
Mucopolysaccharidosis Type I (MPS I)Hurler SyndromeHurler-Scheie Syndrome
Interventions
GENETIC

RGX-111

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

Trial Locations (4)

19104

Children's Hospital of Philadelphia, Philadelphia

92868

Children's Hospital of Orange County, Orange

5265601

Sheba Medical Center, Tel Litwinsky

90035-903

Hospital de Clinicas de Porto Alegre, Porto Alegre

All Listed Sponsors
lead

REGENXBIO Inc.

INDUSTRY

NCT03580083 - RGX-111 Gene Therapy in Patients With MPS I | Biotech Hunter | Biotech Hunter