Status:
ENROLLING_BY_INVITATION
Long Term Follow-up for RGX-202
Lead Sponsor:
REGENXBIO Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
Brief 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 (a gene therapy designed to deliver a transgene for no...
Detailed Description
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) c...
Eligibility Criteria
Inclusion
- 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in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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
- No exclusion criteria apply in this observational follow up study.
Key Trial Info
Start Date :
May 8 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2029
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT06491927
Start Date
May 8 2024
End Date
December 1 2029
Last Update
May 13 2025
Active Locations (2)
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1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
2
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611