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)
REGENXBIO Inc.
Summary
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
Description
Duchenne muscular dystrophy (Duchenne) is a rare genetic disorder, caused by mutations in the gene responsible for making dystrophin, a protein of central importance for muscle cell structure and function. The absence of functional dystrophin protein in individuals with Duchenne results in cell damage during muscle contraction leading to cell death, inflammation, and fibrosis in muscle tissues, and ultimately progressive muscle weakness. RGX-202 is designed to use the AAV8 vector to deliver a transgene to muscle cells that encodes a novel microdystrophin that includes the functional elements o…
Eligibility
- Age range
- 1+ years
- Sex
- Male
- Healthy volunteers
- No
Part 1 - Key Inclusion Criteria: * The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements. * Is a male at least 4 years of age and less than 12 years of age at consent or 1 to \<4 years of age at the time of dosing and ≥ 10 kg at the time of screening. * Must meet any of the following criteria: * DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD w…
Interventions
- GeneticRGX-202
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin
Locations (17)
- Arkansas Children's HospitalLittle Rock, Arkansas
- Stanford School of Medicine /Division of Neuromuscular MedicinePalo Alto, California
- Children's Hospital ColoradoAurora, Colorado
- University of FloridaGainesville, Florida
- Rare Disease ResearchAtlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois