Clinical Trial

Long Term Follow-Up for RGX-111

Enrolling by Invitation
View on ClinicalTrials.gov →
Summary
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2023-10-25; most recent amendment 2026-04-23.
Trial Details
NCT Number NCT06103487
Lead Sponsor REGENXBIO Inc.
Conditions Mucopolysaccharidosis I
Enrollment 21 participants
Start Date 2023-07-24
Primary Completion 2027-09 (estimated)
Study Completion 2027-09 (estimated)
Updated on ClinicalTrials.gov 2026-04-29