200 Participants Needed

Anti-AAV8 Antibody Assessment for Duchenne Muscular Dystrophy

PA
Overseen ByPatient Advocacy
Age: < 65
Sex: Male
Trial Phase: Academic
Sponsor: REGENXBIO Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines boys with Duchenne muscular dystrophy (DMD) to determine if they have antibodies against a specific virus used in gene therapy. These antibodies can influence the effectiveness of certain treatments, making it crucial to identify who has them for future therapies. Boys with DMD who have not participated in a gene therapy study before might be suitable candidates.

As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to significant advancements in DMD therapies.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications.

Why are researchers excited about this trial?

Researchers are excited about the AFFINITY BEYOND trial because it focuses on understanding the role of anti-AAV8 antibodies in boys with Duchenne Muscular Dystrophy (DMD). Unlike treatments that aim to directly modify the disease itself, this study is about screening and assessing immune responses, specifically the presence of antibodies that could affect gene therapy outcomes. By gaining insights into these immune responses, the trial could pave the way for more effective and tailored gene therapies in the future, offering hope for improved management of DMD.

Are You a Good Fit for This Trial?

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks
1 phone/video interview, 1 home health visit

Antibody Testing

Blood sample collected for anti-AAV8 antibody testing

1 day
1 home health visit

Results Communication

Communication of AAV8 antibody test results

1 day
1 phone/video call

Follow-up

Participants are monitored for safety and effectiveness after treatment

90 days

What Are the Treatments Tested in This Trial?

Interventions

  • Observational Screening Study

Find a Clinic Near You

Who Is Running the Clinical Trial?

REGENXBIO Inc.

Lead Sponsor

Trials
25
Recruited
3,100+
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