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AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Boys With DMD

Recruiting
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
Inclusion Criteria
  • 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
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Exclusion Criteria
  • Prior participation in a gene therapy trial OR recipient of a gene therapy drug
  • Other inclusion/exclusion criteria apply
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Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Prevalence of anti-AAV8 antibodies in patients with DMD90 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
NameTimeMethod
Prevalence of anti-AAV9 antibodies in patients with DMD90 days

• To evaluate the prevalence of AAV9 antibodies in patients with DMD

Trial Locations

Locations (1)

Rare Disease Research

🇺🇸

Atlanta, Georgia, United States

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