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

• Sponsor: REGENXBIO Inc. (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05683379
• Collaborator: (none)
• Enrollment: 200
• Location: 1
• Anticipated Duration (Months): 23.4
• Patients Per Site Per Month: 8.5

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy	Diagnostic Test: AAV8 DetectCDx

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

Study Design

• Study Type: Observational
• Anticipated Enrollment: 200 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Anti-AAV8 Antibody Assessment Study of Boys With Duchenne Muscular Dystrophy Aged 0 to <12 Years
• Actual Study Start Date: Dec 20, 2022
• Anticipated Primary Completion Date: Dec 1, 2024
• Anticipated Study Completion Date: Dec 1, 2024

Outcome Measures

Primary Outcome Measures

1. 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

1. Prevalence of anti-AAV9 antibodies in patients with DMD [90 days]

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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 0 Years to 11 Years
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Rare Disease Research	Atlanta	Georgia	United States	30329

Sponsors and Collaborators

• REGENXBIO Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: REGENXBIO Inc.
• ClinicalTrials.gov Identifier: NCT05683379
• Other Study ID Numbers: RGX-202-0101
• First Posted: Jan 13, 2023
• Last Update Posted: Jan 13, 2023
• Last Verified: Jan 1, 2023

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: Yes
• Keywords provided by REGENXBIO Inc.: DMD; Duchenne Muscular Dystrophy; Duchenne
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne

Study Results

No Results Posted as of Jan 13, 2023