Duchenne Muscular Dystrophy
Conditions
Keywords
DMD, Duchenne Muscular Dystrophy, Duchenne
Brief 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).
Detailed 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 results
Interventions
DIAGNOSTIC_TESTAAV8 DetectCDx
An in vitro diagnostic assay to detect antibodies to AAV8 in human serum specimens.
Sponsors
REGENXBIO Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
0 Years to 25 Years
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence of anti-AAV8 antibodies in patients with DMD | 90 days | * To evaluate the prevalence of AAV8 antibodies in patients with DMD * To identify participants who may be eligible for investigational gene therapy clinical trials in males with DMD |
Countries
United States
Outcome results
None listed