Actively Recruiting
A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
Led by REGENXBIO Inc. · Updated on 2025-12-17
65
Participants Needed
17
Research Sites
130 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating RGX-202, a gene therapy designed to deliver a novel microdystrophin protein including important functional elements of naturally occurring dystrophin, for males with Duchenne muscular dystrophy (Duchenne). This rare genetic disorder results in progressive muscle weakness due to the lack of functional dystrophin protein, which is essential for muscle cell structure and function. The study is an open-label, multicenter clinical trial conducted in multiple phases to assess safety, tolerability, pharmacodynamics, pharmacokinetics, and clinical efficacy of this one-time intravenous gene therapy dose. The study is conducted in three parts: Part 1 evaluates two doses of RGX-202 (1x10^14 and 2x10^14 GC/kg) in up to 15 participants to assess safety and tolerability over 52 weeks. Part 2 expands to about 30 participants receiving the higher dose (2x10^14 GC/kg), and Part 3 is a confirmatory study with approximately 30 participants receiving the same dose. All participants receive a single IV infusion of RGX-202 and a short-term immunosuppression regimen to reduce immune response. Participants will be followed and assessed at various time points for up to 104 weeks after dosing, with an option to join a long-term follow-up study per gene therapy safety guidelines. Participants will undergo assessments including safety monitoring through adverse event reporting, pharmacodynamic measurements of microdystrophin protein expression, and pharmacokinetic testing in muscle and serum samples. Functional motor assessments such as Time to Stand, Time to Walk/Run 10 meters, Time to Climb 4 Stairs, North Star Ambulatory Assessment, and Peabody Developmental Motor Scale are conducted at specified intervals. The study also monitors vector shedding and cardiac function. Participants and their guardians will comply with scheduled visits, treatment administration, and study procedures throughout the study duration.
CONDITIONS
Brief Title
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participant
- At least 1 year old and weighing 10 kg or more at screening
- Able to walk independently (10 meters for ages 1 to <4 years; 100 meters for ages 4 years and older) without assistive devices
- Stable dose of systemic glucocorticoids for at least 12 weeks (or consistently on or off steroids for certain cohorts)
- Clinical lab tests including liver and kidney function are normal or not clinically significant
- Documentation of adherence to local vaccination schedules, including willingness to receive meningococcal vaccine if needed
- Willing and able to comply with scheduled visits, study treatment plan, and procedures
- Confirmed Duchenne muscular dystrophy gene mutation (specific exon requirements vary by study part)
You will not qualify if you...
- Any condition contraindicating immunosuppression treatment
- Previous treatment with ataluren or exon-skipping therapy within 6 months or inability to avoid these treatments for 5 years after RGX-202
- Prior receipt of any investigational or commercial gene therapy
- Current or recent (within 3 months) use of other investigational interventions excluding corticosteroids
- Impaired heart function defined as left ventricular ejection fraction less than 55%
- Detectable AAV8 total binding antibodies in serum (Parts 2 and 3)
- Not considered a good candidate for the study by investigator opinion
- For corticosteroid vamorolone users, temporary switch to prednisolone/prednisone around treatment may be required
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day treatment with safety and efficacy monitored up to 52 weeks in Part 1, and up to 104 weeks in Parts 2 and 3
Participants receive a one-time intravenous infusion of RGX-202 gene therapy and a short-term immunosuppression regimen to reduce immune response.
Regular visits for assessments up to 104 weeks after infusion
Duration - Up to 104 weeks after treatment, with the option to enroll in a long-term follow-up study
Participants are monitored for long-term safety, pharmacodynamics, pharmacokinetics, and clinical efficacy after receiving RGX-202.
Multiple visits for assessments including motor function tests and protein expression measurements
Trial Site Locations
Total: 17 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto, California, United States, 94304
Actively Recruiting
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
4
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
5
Rare Disease Research
Atlanta, Georgia, United States, 30329
Actively Recruiting
6
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
7
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
8
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States, 01608
Actively Recruiting
9
Cincinnati Children's
Cincinnati, Ohio, United States, 45229
Actively Recruiting
10
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
11
The University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
12
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States, 23510
Actively Recruiting
13
Children's Hospital of Richmond at Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
14
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A
Not Yet Recruiting
15
BC Children's Hospital
Vancouver, British Columbia, Canada, V65 3N1
Actively Recruiting
16
Children's Hospital London Health Science Centre
London, Ontario, Canada
Actively Recruiting
17
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
Research Team
P
Patient Advocacy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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