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

First listed
Jan 20, 2023
Start date
Jan 4, 2023
Status verified
Jun 2026
Primary completion
Aug 31, 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