REGENXBIO Inc.
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.
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 investig…
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in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from…
Observational study
Arctuva estimate
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