Actively Recruiting

Age: 0Years - 25Years
MALE
ID05683379

Anti-AAV8 Antibody Assessment Study of Males with Duchenne Muscular Dystrophy Aged 0 to <25 Years

Led by REGENXBIO Inc. · Updated on 2025-11-25

200

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are conducting an observational screening study to find out how common anti-adeno-associated serotype 8 (AAV8) antibodies are in males with Duchenne muscular dystrophy (DMD). The goal is to gather information that may help identify candidates for future DMD gene therapy clinical trials. This study is sponsored by REGENXBIO Inc. and focuses on males aged from birth up to 25 years. Participants will take part in interviews either by phone, video, or during a clinic visit to provide consent and share their medical history. They will then have a single blood sample collected either at home by a health professional or during a later clinic visit to test for AAV8 antibodies. After testing, participants will receive their antibody test results through a phone or video call. During the study, researchers will review the antibody test results to determine the presence of anti-AAV8 antibodies within 90 days. The study involves minimal visits and procedures, focusing mainly on blood sample collection and communication of results. Participants are involved for the period needed to complete these steps, with no ongoing treatment provided as part of the study.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 0Years - 25Years
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Males aged 0 to less than 25 years
  • Diagnosis of Duchenne muscular dystrophy
  • Signed and dated informed consent and assent as required by local regulations
Not Eligible

You will not qualify if you...

  • Prior participation in a gene therapy trial or recipient of a gene therapy drug
  • Other inclusion or exclusion criteria may apply

AI-Screening

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Your Study Journey

Screening

Duration - Up to 90 days

Participants are screened for eligibility to participate in the trial.

1 to 2 visits including a phone/video interview or in-clinic visit for consent and medical history, and a home health or in-clinic visit for blood sample collection

Trial Site Locations

Total: 1 location

1

Rare Disease Research

Atlanta, Georgia, United States, 30329

Actively Recruiting

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Research Team

P

Patient Advocacy

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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