AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- NCT05683379
- Lead Sponsor
- REGENXBIO Inc.
- 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 to provide e-consent and medical history
* A single home health visit to collect blood sample for antibody testing
* A phone/video call for communication of AAV8 antibody test results
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 200
- Males at least 0 to <12 years of age
- Diagnosis of DMD
- Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements
- Prior participation in a gene therapy trial OR recipient of a gene therapy drug
- Other inclusion/exclusion criteria apply
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method Prevalence of anti-AAV9 antibodies in patients with DMD 90 days • To evaluate the prevalence of AAV9 antibodies in patients with DMD
Trial Locations
- Locations (1)
Rare Disease Research
🇺🇸Atlanta, Georgia, United States