For Healthcare Providers
AFFINITY DUCHENNE™ is a multicenter, open-label dose escalation and dose expansion study to evaluate the safety, tolerability, and efficacy of a one-time intravenous (IV) dose of RGX-202 in boys with Duchenne, aged 4 to 11 years old.
About RGX-202
- RGX-202 contains an optimized microdystrophin transgene with unique features providing potential benefits as a one-time gene therapy for Duchenne
- RGX-202 is designed to encode a microdystrophin protein that retains key elements of full-length dystrophin including the extended coding region of the C-terminal (CT) domain
- The CT domain recruits several key proteins to the muscle cell membrane1,2
- Dystrobrevin (Dbr)
- Syntrophin (Syn)
- Neuronal Nitric Oxide Synthase (nNOS)
- RGX-202, with an extended CT domain, significantly enhanced elements of the dystrophin associated protein complex at the sarcolemma in mdx mice and increased the half-life of the microdystrophin
- The CT domain recruits several key proteins to the muscle cell membrane1,2
- Increased muscle resistance to lengthening contraction-induced muscle damage in mdx mice after RGX-202 administration2
Current Enrollment Opportunities
Boys with Duchenne, aged 4 to 11 years old, will be enrolled in two dose groups (cohorts). After an independent safety data review, a dose expansion phase of the study allows for up to six additional boys to be enrolled in each group. This would be up to nine boys in each group, or 18 boys total.
Eligibility
Below are key eligibility criteria for the AFFINITY DUCHENNE Study. For a more complete list of eligibility criteria, a list of AFFINITY DUCHENNE Study locations, and more information, visit ClinicalTrials.gov (identifier #NCT05693142).
Key Eligibility Criteria
Eligible participants must be males aged 4 to 11 years of age on Day 1 of study and have a documented diagnosis of Duchenne. For a full list of eligibility criteria and additional information, please visit clinicaltrials.gov.
If you have any patients in your practice who may be eligible to participate in the AFFINITY DUCHENNE study, we encourage you to contact a study site.
Primary study Endpoints
Participants will be assessed (safety) for 52 weeks following treatment with RGX-202 to evaluate incidences of adverse events (AEs) and serious adverse events (SAEs).
Secondary study Endpoints
Efficacy measured by change in functional assessment as measured by North Star Ambulatory Assessment (NSAA) raw and total score
Time Frame: Multiple timepoints through 52 weeks
Microdystrophin protein expression:
RGX-202 microdystrophin protein levels determined in muscle biopsy and vector genome concentrations in muscle
Time Frame: 12 weeks
Pharmacokinetics:
Vector genome concentrations as measured by polymerase chain reaction (PCR) to RGX-202 DNA in serum
Time Frame: Multiple timepoints through 52 weeks
Vector Shedding:
Vector genome concentrations as measured by PCR to RGX-202 DNA in urine
Time Frame: Multiple timepoints through 52 weeks
For more information about the study, please contact medinfo@regenxbio.com or visit ClinicalTrials.gov (identifier #NCT05693142).
Anti-AAV8 Antibody Assessment Study of Boys with Duchenne muscular dystrophy (DMD)
REGENXBIO is also currently recruiting for an observational study called AFFINITY BEYONDTM to understand how common antibodies against AAV8 are in people with Duchenne.
The study may enroll up to 200 U.S. participants.
Key eligibility criteria include:
- Boys 0 to 11 years old
- Diagnosis of Duchenne muscular dystrophy
- Has not received any investigational or commercial gene therapy product over his lifetime
Parents and Families
If you would like to contact a member of our Patient Advocacy team, please email us at Duchenne@regenxbio.com Additional information and study site contact details can be found at ClinicalTrials.gov (identifier #NCT05693142).
Healthcare Providers
If you are a healthcare provider and would like more information on our study, please contact us at medinfo@regenxbio.com.
