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Enrolling by invitation NCT06491927

Long Term Follow-up for RGX-202

— Observational Duchenne Muscular Dystrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: No Intervention.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: No limits · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)

Overview

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.

Detailed description

This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of investigational RGX-202. Eligible participants are those who have undergone evaluation in a previous (parent) clinical study following a single intravenous infusion of RGX-202 for the treatment of DMD. Enrollment in the current long-term follow-up (LTFU) study will occur after the participant has completed or discontinued from the parent study. Participants will be followed in this study cumulatively for up to 5 years after RGX-202 administration (inclusive of the parent study). No investigational treatment will be administered under this protocol. The total study duration for each participant may vary depending on when he enrolls in the current study following RGX-202 administration in the parent study.

Interventions

  • Genetic No Intervention
    Observational study

Primary outcome measures

  • Evaluation of long-term safety of RGX-202 [Time frame: Baseline, 5 years inclusive of parent study]
Secondary outcome measures (9)
  • Time to Stand (TTSTAND) [Time frame: Baseline, 5 years inclusive of parent study]
  • Time to Run/Walk (TTRW) [Time frame: Baseline, 5 years inclusive of parent study]
  • Time to Climb (TTCLIMB) [Time frame: Baseline, 5 years inclusive of parent study]
  • North Start Ambulatory Assessment (NSAA) [Time frame: Baseline, 5 years inclusive of parent study]
  • Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Control Subtest [Time frame: Baseline, 5 years inclusive of parent study]
  • Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Transport Subtest [Time frame: Baseline, 5 years inclusive of parent study]
  • Stride velocity 95th centile (SV95C) [Time frame: Baseline, 5 years inclusive of parent study]
  • Pharmacokinetics (PK) [Time frame: Baseline, 5 years inclusive of parent study]
  • Vector Shedding [Time frame: Baseline, 5 years inclusive of parent study]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

United States · 5 centers
  • Arkansas Children's Hospital — Little Rock
  • Stanford School of Medicine /Division of Neuromuscular Medicine — Palo Alto
  • Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
  • The University of Texas Southwestern Medical Center — Dallas
  • Virginia Commonwealth University — Richmond

Identifiers

NCT: NCT06491927 · RGX-202-5101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗