Long Term Follow-up for RGX-202

Launched by REGENXBIO INC. · Jul 1, 2024

Keywords

ClinConnect Summary

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 treat...

Gender

MALE

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a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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