NCT06491927Enrolling by invitation
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)
Source: ClinicalTrials.gov · Last updated 24 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Study type
- Observational
- Enrollment
- 66 (estimated)
- First submitted
- 1 Jul 2024
- Start date
- 8 May 2024
- Primary completion date
- 1 Dec 2029
- Lead sponsor
- REGENXBIO Inc.
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT06491927
What you commit to
- Study type: Observational
- Planned participants: 66
- From 8 May 2024 to the primary completion date, 1 Dec 2029
- 5 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- No exclusion criteria apply in this observational follow up study.
Who the study is looking for
- 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in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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.
Sex: Male · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Ann & Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| The University of Texas Southwestern Medical Center Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| Arkansas Children's Hospital Coordinator of this site? Confirm its status | Little Rock | United States | Unknown status |
| Stanford School of Medicine /Division of Neuromuscular Medicine Coordinator of this site? Confirm its status | Palo Alto | United States | Unknown status |
| Virginia Commonwealth University Coordinator of this site? Confirm its status | Richmond | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.