Actively Recruiting
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
Led by REGENXBIO Inc. · Updated on 2025-12-17
65
Participants Needed
17
Research Sites
290 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne. For additional information on how to participate (or be considered for the study), please follow this link: https://mytomorro.ws/affinity-ct-gov
CONDITIONS
Official 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 aged at least 1 year and weighing 10 kg or more at screening
- For Part 1: male participants at least 4 years but less than 12 years at consent or 1 to less than 4 years at dosing
- DMD gene mutation as defined: exons 18 and above or exons 12-17 for certain cohorts
- Ability to walk independently: 100 meters if 4 years and older, 10 meters if 1 to less than 4 years (with some exceptions for cohorts)
- Ability to complete the TTSTAND test per protocol
- Stable dose of systemic glucocorticoids for at least 12 weeks according to standard care, or consistent on/off for younger participants
- Clinical laboratory tests including liver and kidney function within normal limits or not clinically significant
- Documentation of adherence to local vaccination schedules and willingness to receive meningococcal vaccine if needed
- Willingness and ability of participant and guardian to comply with study visits, treatment, and procedures
You will not qualify if you...
- Any condition that contraindicates immunosuppression treatment
- Receipt of ataluren or exon-skipping therapy within 6 months prior to study or inability to refrain from these therapies for 5 years after RGX-202
- Prior receipt of any investigational or commercial gene therapy
- Current or recent use (within 3 months) of other investigational interventions except corticosteroids
- Impaired cardiac function defined as left ventricular ejection fraction less than 55%
- Detectable AAV8 total binding antibodies in serum (for Part 2 and 3)
- Participant deemed not a good candidate for the study by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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