Long Term Follow-up for RGX-202

NCT06491927 | Enrolling By Invitation FDA Drug

• 1 other identifier: RGX-202-5101
• Study Type: observational
• Target: 66
• Locations: 1 country
• Sites: 5

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 (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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Gene therapy; DMD; Duchenne; Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Evaluation of long-term safety of RGX-202

  Incidences of AEs and SAEs over time

  Baseline, 5 years inclusive of parent study

Secondary Outcomes (9)

• Time to Stand (TTSTAND)

  Baseline, 5 years inclusive of parent study

• Time to Run/Walk (TTRW)

  Baseline, 5 years inclusive of parent study

• Time to Climb (TTCLIMB)

  Baseline, 5 years inclusive of parent study

• North Start Ambulatory Assessment (NSAA)

  Baseline, 5 years inclusive of parent study

• Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Control Subtest

  Baseline, 5 years inclusive of parent study

Study Arms (1)

RGX-202 Recipients

Subjects who have received RGX-202 in a separate parent study.

Genetic: No Intervention

Interventions

No Intervention GENETIC

Observational study

RGX-202 Recipients

Eligibility Criteria

• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Received RGX-202 in a separate parent study.

You may qualify if:

• 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in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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.

Sponsors & Collaborators

• REGENXBIO Inc.: lead

Study Sites (5)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Stanford School of Medicine /Division of Neuromuscular Medicine

Palo Alto, California, 94304, United States

Location

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

Chicago, Illinois, 60611, United States

Location

The University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2024
• First Posted: July 9, 2024
• Study Start: May 8, 2024
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: March 24, 2026

Record last verified: 2026-03

Locations

US (5)