Evaluating AAV8 Antibodies in Boys with Duchenne Muscular Dystrophy
Anti-AAV8 Antibody Assessment Study of Males With Duchenne Muscular Dystrophy Aged 0 to <25 Years
This study is testing how common certain antibodies are in boys with Duchenne muscular dystrophy to help find out who might be able to join future gene therapy trials.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Ages | 0 Years to 25 Years |
| Sex | Male |
| Sponsor | REGENXBIO Inc. Industry-sponsored |
| Locations | 1 site (Atlanta, Georgia) |
| Trial ID | NCT05683379 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in boys diagnosed with Duchenne muscular dystrophy (DMD). Participants will undergo a phone or video interview for consent and medical history, followed by a home health visit to collect blood samples for antibody testing. The results may help identify potential candidates for future gene therapy clinical trials targeting DMD.
Who should consider this trial
Good fit: Ideal candidates are males aged 0 to under 12 years with a confirmed diagnosis of Duchenne muscular dystrophy.
Not a fit: Patients who have previously participated in a gene therapy trial or received a gene therapy drug may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance the identification of suitable candidates for innovative gene therapies in DMD.
How similar studies have performed: While this study focuses on antibody prevalence, similar approaches in gene therapy trials have shown promise in identifying suitable candidates.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Males at least 0 to \<25 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
Where this trial is running
Atlanta, Georgia
- Rare Disease Research — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Study coordinator: Patient Advocacy
- Email: Duchenne@regenxbio.com
- Phone: (833) 711-0349
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.