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.
Eligibility
Sex
MALE
Min age
—
Max age
—
Healthy volunteers
No
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in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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.
Exclusion Criteria:
* No exclusion criteria apply in this observational follow up study.
Primary outcome measure(s)
Evaluation of long-term safety of RGX-202 — Baseline, 5 years inclusive of parent study Incidences of AEs and SAEs over time
Trial sites (5)
Facility
City
Region
Status
Arkansas Children's Hospital
Little Rock
Arkansas
Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto
California
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Illinois
The University of Texas Southwestern Medical Center
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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