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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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
No Placebo-Only Group
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.
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.
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Safety measured by incidence of Adverse Events and Serious Adverse Events
Secondary study objectives
Efficacy measured by change in Functional Assessment
Microdystrophin protein expression
Pharmacokinetics (PK)
+1 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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
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Who is running the clinical trial?
REGENXBIO Inc.Lead Sponsor
24 Previous Clinical Trials
2,730 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been on a stable dose of steroids for at least 12 weeks before my recent tests.I can walk 100 meters on my own without help.I do not have any conditions that prevent me from taking immunosuppressive drugs.I have taken ataluren or exon-skipping therapy for DMD within the last 6 months or cannot stop taking it for 5 years after starting RGX-202.My liver and kidney tests are normal or not concerning.My heart's pumping ability is below normal.My DMD is caused by a mutation in exon 18 or higher.I can complete the required standing test as per the study's guidelines.
Research Study Groups:
This trial has the following groups:- Group 1: RGX-202 Dose 2
- Group 2: RGX-202 Dose 1
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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