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Enrolling by invitation Observational

Long Term Follow-up for RGX-202

NCT06491927 · tracked via the Priya Life Science USA tracker
Phase
Observational
Started
2024-05-08
Last updated
2026-03-24

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

No Intervention

No Intervention: Observational study

Study 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.

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a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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

FacilityCityRegionStatus
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 Dallas Texas
Virginia Commonwealth University Richmond Virginia
Official registry record

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.

View NCT06491927 on ClinicalTrials.gov ↗ ← All trials in the USA