Long Term Follow-up for RGX-202

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Genetic: No Intervention

• Registration Number: NCT06491927
• Lead Sponsor: REGENXBIO Inc.

Brief 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 (a 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 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.

Study Timeline

• Study Start: 2024-05-08
• Status Verified: 2024-07
• Study First Submitted: 2024-07-01
• Study First Submitted QC: 2024-07-01
• Last Update Submitted: 2024-07-01
• Study First Posted: 2024-07-09
• Last Update Posted: 2024-07-09
• Primary Completion: 2029-12-01
• Study Completion: 2029-12-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: Male
• Target Recruitment: 19

Inclusion Criteria

• The parent(s) or legal guardian(s) of the participant has/(have) provided written informed consent and (where applicable) Health Insurance Portability and Accountability Act (HIPAA) authorization after the nature of the study has been explained, prior to any research-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.
• Must have undergone evaluation in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.
• Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, and study procedures.
• Sexually active participants must be willing to use a medically accepted method of contraception from the time of the screening visit through 5 years after RGX-202 administration.

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Exclusion Criteria

• No exclusion criteria apply in this observational follow up study.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
RGX-202 Recipients	No Intervention	Subjects who have received RGX-202 in a separate parent study.

Primary Outcome Measures

Name	Time	Method
Evaluation of long-term safety of RGX-202	5 years inclusive of parent study	Incidences of AEs and SAEs over time

Secondary Outcome Measures

Name	Time	Method
Efficacy measured by change in Functional Assessment	5 years inclusive of parent study	Longitudinal trajectory (mean and change from baseline) in North Star Ambulatory Assessment (NSAA) raw and linear score

Trial Locations

Locations (3)

Stanford School of Medicine/Division of Neuromuscular Medicine; 🇺🇸 San Carlos, California, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States