Long-term follow-up of people treated with RGX-202 for Duchenne muscular dystrophy
Follows people with Duchenne muscular dystrophy who already received RGX-202 gene therapy.
Plain-language title generated from the registry record
Registry title: Long Term Follow-up for RGX-202
Key facts
- Where
- Chicago, Dallas, Little Rock, Palo Alto, and Richmond, United StatesSee the 5 sites
Where in United States
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesNo longer taking part
- The University of Texas Southwestern Medical CenterDallas, United StatesNo longer taking part
- Arkansas Children's HospitalLittle Rock, United StatesNo longer taking part
- Stanford School of Medicine /Division of Neuromuscular MedicinePalo Alto, United StatesNo longer taking part
- Virginia Commonwealth UniversityRichmond, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- REGENXBIO Inc.
- Trial code
- NCT06491927
- Record updated
- 24 March 2026
- Original description
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.Read full original descriptionShow less
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
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.
Inclusion: 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.
Exclusion: No exclusion criteria apply in this observational follow up study.