Assessing Pre-existing Antibodies to AAV in a Population of Patients With PKP2-Associated ARVC

Overview

About this study

The purpose of this multicenter, seroprevalence study to assess pre-existing antibodies to Adenovirus-associated Virus Vector (AAV) in a population of patients with Plakophilin 2 (PKP2)-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), who may be eligible for subsequent gene therapy trials. Biological samples and clinical data will be collected over a period of up to 5 years from the date of enrollment. No investigational product will be administered.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria: 

  • Ages 14 to 65 years, inclusive, at the time of consent.
  • Pathogenic or likely pathogenic Plakophilin 2 (PKP2) gene mutation.
  • Diagnosed with Arrhythmogenic Right Ventricular Cardiomyopathy (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.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 4/19/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

John Giudicessi, M.D., Ph.D.

Open for enrollment

Contact information:

CVRU Heart Rhythm Services Research Team

(507) 255-0774

More information

Publications

Publications are currently not available