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RGX-202 Recipients for Duchenne Muscular Dystrophy

N/A
Waitlist Available
Research Sponsored by REGENXBIO Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years inclusive of parent study
Awards & highlights

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 novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years inclusive of parent study
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years inclusive of parent study for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Evaluation of long-term safety of RGX-202
Secondary study objectives
Efficacy measured by change in Functional Assessment

Trial Design

1Treatment groups
Experimental Treatment
Group I: RGX-202 RecipientsExperimental Treatment1 Intervention
Subjects who have received RGX-202 in a separate parent study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
No Intervention
2017
Completed Phase 1
~4810

Find a Location

Who is running the clinical trial?

REGENXBIO Inc.Lead Sponsor
24 Previous Clinical Trials
2,531 Total Patients Enrolled
~13 spots leftby Dec 2029
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