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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
14 Years to 65 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 5 years
  • Procedures mentioned: Questionnaires

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Clinical and observational data will be collected prospectively for up to 5 years from the date of enrollment, or until the patient withdraws consent/assent, undergoes heart transplantation, or dies."
  • "Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Official eligibility 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.

Study sites (8)

CA

CO

  • University of Colorado, Denver · Aurora, CO 80045Matthew Taylor, MD, PhD · Study contactMore trials in Aurora

MA

MD

  • John Hopkins University School of Medicine · Baltimore, MD 21287Hugh G Calkins, MD · Study contactMore trials in Baltimore

MN

NY

OH

SC