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Arrhythmogenic Cardiomyopathy (ACM) Patient Attitudes Toward Gene Therapy Trial Design

🕛 This survey takes about 10 minutes to complete.

❓ For any questions while taking this survey, please reach out to support@fiacardiac.org.

Purpose of This Survey

Researchers design clinical trials to answer important questions about whether a new treatment is safe and works better than current care. To do this fairly, they may use several different approaches. In this survey, we would like to understand how people affected by ACM (also known as ARVC) view two possible ways of designing a gene therapy clinical trial. We are asking only about trial design. Your responses will help us understand which approach patients and families find more acceptable and why.

This survey is anonymous. It does not collect your name, email, or any identifying information, and only de-identified summaries will ever be shared.

Completing the survey does not affect access to a clinical trial or treatment. Because these conditions are genetic or inherited, there may be multiple affected family members. If you are affected and also have a child (or children) who are affected, you have the opportunity to take the survey for yourself and for your affected child(ren). The adeno-associated virus (AAV) gene therapy is investigational, meaning it has not yet been proven safe or effective. It is dosed (given) only one time.

Potential Benefits

May address the underlying genetic cause of disease

May slow disease progression (i.e., slow the disease from getting worse, improve function and/or heart rhythm, or prevent serious events)

May provide long-lasting effects after a single treatment

Potential Risks

Immune or infusion-related reactions

Other side effects may occur due to gene therapy or immunosuppression

Long-term safety and effectiveness are still being studied

The survey is being distributed by FIA in partnership with Tenaya Therapeutics, which is sponsoring the survey. FIA will analyze the anonymous feedback and provide a de-identified summary to Tenaya. The goal of this survey is to improve the experience of patients participating in clinical trials of gene therapies, while also maintaining scientific integrity.