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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

Anti-AAV8 Antibody Assessment Study of Boys With Duchenne Muscular Dystrophy Aged 0 to <12 Years

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05683379
• Other study ID #: RGX-202-0101
• Status: Recruiting
• Start date: December 20, 2022
• Est. completion date: December 2024

Study information

• Verified date: January 2023
• Source: REGENXBIO Inc.
• Contact: Patient Advocacy
• Phone: 866-860-0117
• Email: Duchenne[@]regenxbio.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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 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 information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 0 Years to 11 Years

Eligibility

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

Exclusion Criteria:

• Prior participation in a gene therapy trial OR recipient of a gene therapy drug

• Other inclusion/exclusion criteria apply

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Diagnostic Test:
• AAV8 DetectCDx
An in vitro diagnostic assay to detect antibodies to AAV8 in human serum specimens.

Locations

Country	Name	City	State
United States	Rare Disease Research	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
REGENXBIO Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence of anti-AAV8 antibodies in patients with DMD	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	90 days
Secondary	Prevalence of anti-AAV9 antibodies in patients with DMD	• To evaluate the prevalence of AAV9 antibodies in patients with DMD	90 days