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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of California San Francisco
  San Francisco, California, United States Recruiting
• University of Colorado, Denver
  Aurora, Colorado, United States Recruiting
• John Hopkins University School of Medicine
  Baltimore, Maryland, United States Recruiting
• Brigham and Women's Hospital
  Boston, Massachusetts, United States Recruiting
• Mayo Clinic
  Rochester, Minnesota, United States Recruiting
• New York University
  New York, New York, United States Recruiting

Study contacts

• Study coordinator: Matthew Pollman, MD
• Email: mpollman@tenayathera.com
• Phone: 650-209-8092

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• FAPI PET/CT imaging in arrhythmogenic right ventricular cardiomyopathy

  This study will test whether a PET/CT scan using a 68Ga-DOTA-SA-FAPI tracer can detect active scarring in adults with ARVC and provide information beyond standard heart imaging.

  Recruiting Istanbul University - Cerrahpasa · Istanbul, Fatih · NCT07563660

• Gene therapy for adults with PKP2 mutation-associated heart disease

  This study is testing a new gene therapy called TN-401 to see if it can help adults with heart disease caused by a specific genetic mutation feel better and stay safe.

  Recruiting at 7 sites Phase 1 Tenaya Therapeutics · San Francisco, California and 6 other locations · NCT06228924

• Targeted therapy for arrhythmogenic cardiomyopathy using tideglusib

  This study is testing a new medication called tideglusib to see if it can help people with a specific genetic type of arrhythmogenic cardiomyopathy feel better and reduce their risk of serious heart problems.

  Recruiting at 10 sites Phase 2 Hamilton Health Sciences Corporation · Calgary, Alberta and 16 other locations · NCT06174220

• Examining exercise effects on heart activity in ARVC patients

  This study tests how different types of exercise affect heart activity in people with Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) to help create safer exercise guidelines for them.

  Recruiting Swiss Federal Institute of Technology · Zurich, Canton of Zurich · NCT06040242

• Registry for patients with cardiomyopathies

  This study is collecting health information and samples from people with different types of heart muscle diseases to better understand their causes and effects over time.

  Recruiting University Hospital Heidelberg · Heidelberg, Baden-Wurttemberg · NCT04265040

• Registry and biobank for arrhythmogenic ventricular cardiomyopathy

  This study is trying to gather information and samples from people with arrhythmogenic ventricular cardiomyopathy and their families to better understand the genetic causes of the condition and improve early diagnosis for those at risk.

  Recruiting at 2 sites Mayo Clinic · Rochester, Minnesota and 1 other locations · NCT03049254

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.