Anti-AAV8 Antibody Assessment Study of Males With Duchenne Muscular Dystrophy Aged 0 to <25 Years
REGENXBIO Inc.
Summary
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
Description
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD). Information collected in this study may be used to identify potential participants for DMD investigational gene therapy clinical trials. This study consists of: * A phone/video interview or at an in-clinic visit to provide e-consent and medical history * A single home health visit or subsequent in-clinic visit to collect blood sample for antibody testing * A phone/video call for communication of AAV8 antibody test re…
Eligibility
- Age range
- 0–25 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * Males at least 0 to \<25 years of age * Diagnosis of DMD * Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements Exclusion Criteria: * Prior participation in a gene therapy trial OR recipient of a gene therapy drug * Other inclusion/exclusion criteria apply
Interventions
- Diagnostic TestAAV8 DetectCDx
An in vitro diagnostic assay to detect antibodies to AAV8 in human serum specimens.
Location
- Rare Disease ResearchAtlanta, Georgia