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Gene Therapy

RGX-202 Gene Therapy for Duchenne Muscular Dystrophy

Phase 1 & 2
Recruiting
Research Sponsored by REGENXBIO Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD
Participant is able to walk 100 meters independently without assistive devices, as assessed at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights

Study Summary

This trial tests a new gene therapy to help treat Duchenne muscular dystrophy, testing safety, tolerance and effectiveness of a one-time IV dose.

Who is the study for?
This trial is for individuals with Duchenne Muscular Dystrophy (DMD) who have a specific gene mutation, can walk 100 meters without help, and have been on stable glucocorticoids for at least 12 weeks. They must not have had other gene therapies or certain DMD treatments recently and should not need to avoid immunosuppression.Check my eligibility
What is being tested?
The trial tests RGX-202, a one-time IV gene therapy aimed at delivering a microdystrophin transgene to treat DMD. It's an open-label study evaluating the safety, tolerability, and effectiveness of this new treatment in participants.See study design
What are the potential side effects?
While specific side effects are not listed here, potential risks may include reactions related to immune response against the therapy or complications from intravenous administration. The impact on liver and kidney function will also be monitored.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My DMD is caused by a mutation in exon 18 or higher.
Select...
I can walk 100 meters on my own without help.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety measured by incidence of Adverse Events and Serious Adverse Events
Secondary outcome measures
Efficacy measured by change in Functional Assessment
Microdystrophin protein expression
Pharmacokinetics (PK)
+1 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: RGX-202 Dose 2Experimental Treatment1 Intervention
A single IV infusion of RGX-202 at a dose of 2x10^14 GC/kg body weight
Group II: RGX-202 Dose 1Experimental Treatment1 Intervention
A single IV infusion of RGX-202 at a dose of 1×10^14 GC/kg body weight

Find a Location

Who is running the clinical trial?

REGENXBIO Inc.Lead Sponsor
23 Previous Clinical Trials
2,517 Total Patients Enrolled

Media Library

RGX-202 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05693142 — Phase 1 & 2
Duchenne Muscular Dystrophy Research Study Groups: RGX-202 Dose 1, RGX-202 Dose 2
Duchenne Muscular Dystrophy Clinical Trial 2023: RGX-202 Highlights & Side Effects. Trial Name: NCT05693142 — Phase 1 & 2
RGX-202 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05693142 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the capacity of the study in terms of participants?

"Correct. Clinicaltrials.gov data suggests that this medical trial, posted on January 1st 2023, is actively recruiting patients for inclusion. 18 participants need to be found from one particular site."

Answered by AI

Is this medical experiment open to elderly individuals aged 75 and up?

"This clinical trial is intended for children with ages ranging from 4 to 11. Additionally, there are 61 studies available to minors and 52 research opportunities open to senior citizens."

Answered by AI

Are there vacancies available for potential participants of this trial?

"Affirmative. Data hosted on clinicaltrials.gov confirms that this medical research, which was originally uploaded on the first day of January in 2023, is actively recruiting volunteers. Approximately 18 participants need to be recruited from 1 distinct site."

Answered by AI

Do I meet the requirements to partake in this scientific investigation?

"This medical trial seeks to recruit 18 children between the ages of 4 and 11 who suffer from Duchenne muscular dystrophy. To be eligible, patients must possess a DMD gene mutation in exons 18 or above; they also need to be able to complete certain physical tasks without any assistive devices, have been taking standard glucocorticoid doses for at least 12 weeks prior to screening, and display normal laboratory tests results."

Answered by AI
~11 spots leftby Dec 2025