NCT06491927

Long Term Follow-up for RGX-202

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 66 (estimated)
• Sponsor: REGENXBIO Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.

Detailed description

This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of investigational RGX-202. Eligible participants are those who have undergone evaluation in a previous (parent) clinical study following a single intravenous infusion of RGX-202 for the treatment of DMD. Enrollment in the current long-term follow-up (LTFU) study will occur after the participant has completed or discontinued from the parent study. Participants will be followed in this study cumulatively for up to 5 years after RGX-202 administration (inclusive of the parent study). No investigational treatment will be administered under this protocol. The total study duration for each participant may vary depending on when he enrolls in the current study following RGX-202 administration in the parent study.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
GENETIC	No Intervention	Observational study

Timeline

• Start date: 2024-05-08
• Primary completion: 2029-12-01
• Completion: 2029-12-01
• First posted: 2024-07-09
• Last updated: 2026-03-24

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study