Long Term Follow-up for RGX-202

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

19 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-05-08

Study Completion Date

2029-12-01

Brief Summary

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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 (a 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.

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Detailed Description

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This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of 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.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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RGX-202 Recipients

Subjects who have received RGX-202 in a separate parent study.

No Intervention

Intervention Type GENETIC

Observational study

Interventions

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No Intervention

Observational study

Intervention Type GENETIC

Eligibility Criteria

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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.

Eligible Sex

MALE

Accepts Healthy Volunteers

No