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ENROLLING BY INVITATION
NCT06491927

Long Term Follow-up for RGX-202

Sponsor: REGENXBIO Inc.

View on ClinicalTrials.gov

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.

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

Key Details

Gender

MALE

Age Range

Any - Any

Study Type

OBSERVATIONAL

Enrollment

66

Start Date

2024-05-08

Completion Date

2029-12-01

Last Updated

2026-03-24

Healthy Volunteers

No

Interventions

GENETIC

No Intervention

Observational study

Locations (5)

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Stanford School of Medicine /Division of Neuromuscular Medicine

Palo Alto, California, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Virginia Commonwealth University

Richmond, Virginia, United States