AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
REGENXBIO Inc.
Study ID
NCT05693142
Phase
PHASE2/PHASE3
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
MALE
Age
1 Year - N/A
Healthy Volunteers
Not accepted

Interventions

  • RGX-202 — GENETIC
    RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin

Study Details

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

Key Dates

Start date
Jan 4, 2023
Status verified
Dec 2025
Primary completion
Feb 28, 2026
Completion
Aug 31, 2028

Study Design

Enrollment
65 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1: Cohort 1 and 1b: RGX-202 Dose 1
    A single IV infusion of RGX-202 at a dose of 1×10\^14 GC/kg body weight
  • Experimental: Part 1: Cohort 2, 2c;, and Part 2; and Part 3: RGX-202 Dose 2
    A single IV infusion of RGX-202 at a dose of 2x10\^14 GC/kg body weight

Primary Outcome Measure

Part 1 Safety measured by incidence of Adverse Events and Serious Adverse Events [ Time Frame: 52 weeks ]

Central Contacts

Locations (13)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202
Amber Evans
[email protected]
501-364-2259
Aravindhan Veerapandiyan, MD (PRINCIPAL_INVESTIGATOR)
Stanford School of Medicine /Division of Neuromuscular MedicinePalo AltoCalifornia94304
[email protected]
650-725-4341
Carolina Tesi-Rocha, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital ColoradoAuroraColorado80045
Michele Yang
[email protected]
720-777-6895
Hannah Kleiner
[email protected]
720-777-1377
Michele Yang, MD (PRINCIPAL_INVESTIGATOR)
University of FloridaGainesvilleFlorida32610
Melissa Lewis
[email protected]
352-273-8218
Barry Byrne, MD (PRINCIPAL_INVESTIGATOR)
Rare Disease ResearchAtlantaGeorgia30329
Lily Goggans
[email protected]
470-600-9134
Maureen Ikpeoha
[email protected]
470-666-1884
Han Phan, MD (PRINCIPAL_INVESTIGATOR)
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Nicole Geanous
[email protected]
312-227-3019
Nancy L Kuntz, MD (PRINCIPAL_INVESTIGATOR)
University of IowaIowa CityIowa52242
Laura Knosp
[email protected]
319-384-6277
Dimah Saade, MD (PRINCIPAL_INVESTIGATOR)
University of Massachusetts Chan Medical SchoolWorcesterMassachusetts01608
Tyler Mola
[email protected]
978-503-9219
Eleonora D'Ambrosio, MD (PRINCIPAL_INVESTIGATOR)
Cincinnati Children'sCincinnatiOhio45229
Angela Edmondson
[email protected]
513-636-9285
Cuixia Tian, MD (PRINCIPAL_INVESTIGATOR)
Oregon Health & Science UniversityPortlandOregon97239
Beata Dyar
[email protected]
503-494-8216
Erika Finanger, MD (PRINCIPAL_INVESTIGATOR)
The University of Texas Southwestern Medical CenterDallasTexas75390
Elaine Most
[email protected]
214-456-2463
Susan Iannaccone, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of the King's DaughtersNorfolkVirginia23510
Erika Paradiso
[email protected]
757-668-6981
Crystal Proud, MD (PRINCIPAL_INVESTIGATOR)
Children's Hospital of Richmond at Virginia Commonwealth UniversityRichmondVirginia23298
Falgun Patel
[email protected]
(804) 827-9731
Amy Harper, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Arkansas Children's Hospital· Little Rock, ARStanford School of Medicine /Division of Neuromuscular Medicine· Palo Alto, CAChildren's Hospital Colorado· Aurora, COUniversity of Florida· Gainesville, FLRare Disease Research· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, IL

Related Studies