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Observing the prevalence of antibodies in patients with a specific heart condition

Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

Observational Tenaya Therapeutics · NCT06311708

This study looks at how common certain antibodies are in people with a heart condition called ARVC who have a specific gene mutation, while they receive regular care and share health information over five years.

Quick facts

Study typeObservational
Enrollment200 (estimated)
Ages14 Years to 65 Years
SexAll
SponsorTenaya Therapeutics Industry-sponsored
Drugs / interventionschemotherapy
Locations21 sites (San Francisco, California and 20 other locations)
Trial IDNCT06311708 on ClinicalTrials.gov

What this trial studies

This multicenter, non-interventional study aims to observe the natural progression of Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) in patients with PKP2 gene mutations. Participants will receive standard care while biologic samples are collected annually to measure cardiac biomarkers and assess quality of life. The study will gather clinical and observational data for up to five years, with a focus on the prevalence of pre-existing antibodies against AAV9, which is relevant for potential gene therapy. Patients are encouraged to participate regardless of their interest in gene therapy.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 14-65 with a pathogenic PKP2 gene mutation diagnosed with ARVC.

Not a fit: Patients currently receiving immunosuppressive therapy or with a history of significant liver disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of ARVC and inform future gene therapy approaches.

How similar studies have performed: While similar studies have explored gene therapy and ARVC, this specific observational approach focusing on antibody prevalence is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Ages 14-65 years, inclusive, at the time of consent
* Pathogenic or likely pathogenic PKP2 gene mutation
* Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.
* Functioning ICD

Exclusion Criteria:

* Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
* History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
* Previously dosed with any investigational or approved gene therapy product at any time
* Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.
* History of cardiac transplant.

Where this trial is running

San Francisco, California and 20 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Arrhythmogenic Right Ventricular CardiomyopathyPKP2 Mutation Arrhythmogenic Right Ventricular CardiomyopathyArrhythmogenic CardiomyopathyPKP2-associated ARVCPKP2-ARVCPKP2-ACMAdeno Associated VirusAdeno-Associated Virus Serotype 9
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.