Actively Recruiting
Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Led by Tenaya Therapeutics · Updated on 2024-11-01
200
Participants Needed
21
Research Sites
14 weeks
Total Duration
On this page
Sponsors
T
Tenaya Therapeutics
Lead Sponsor
U
University of California, San Francisco
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting a multicenter, non-interventional observational study to understand the natural progression of Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) in patients with PKP2 gene mutations. The study also aims to assess how common pre-existing antibodies to the AAV9 virus, which is used in gene therapy, are in this patient population. Participation is encouraged for all patients, regardless of their interest in or eligibility for gene therapy. Patients will continue their usual medical care under their healthcare providers. The study involves collecting biological samples annually to measure heart-related and other biomarkers, alongside prospective clinical and observational data collection for up to five years or until events such as heart transplantation or death occur. There may also be a one-time genetic sample collection for research if consent is given. Participants will complete Quality of Life questionnaires, and if not part of their standard care, yearly heart rhythm monitoring using Holter or equivalent devices will be performed. No experimental treatments will be given during this study. Participants will be observed both retrospectively and prospectively. Researchers will review clinical data, imaging, and biomarker results, focusing on antibody presence, illness burden, arrhythmic risk, heart function, and patients' quality of life over five years. The study includes regular assessments and monitoring to gather comprehensive data on disease progression and patient wellbeing, with no additional interventions beyond standard care.
CONDITIONS
Brief Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ages 14 to 65 years at the time of consent
- Have a pathogenic or likely pathogenic mutation in the PKP2 gene
- Diagnosed with Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) meeting 2010 Modified Task Force Criteria
- Have a functioning implantable cardioverter defibrillator (ICD)
You will not qualify if you...
- Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy
- History of significant liver disease, hepatitis B or C, HIV, or tuberculosis infection
- Previously treated with any investigational or approved gene therapy
- Taking part in another interventional clinical trial unless approved by the sponsor
- History of heart transplant
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 5 years
Participants who undergo routine care are observed. Biologic samples are collected annually to measure cardiac and other related biomarkers. Clinical and observational data are collected prospectively for up to 5 years. Quality of Life questionnaires and annual diagnostic Holter (or equivalent) monitoring may be performed if not part of routine care. A one-time genetic sample collection may occur if consent is provided.
Annual visits for sample collection and assessments
Trial Site Locations
Total: 21 locations
1
University of California San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
University of Colorado, Denver
Aurora, Colorado, United States, 80045
Actively Recruiting
3
John Hopkins University School of Medicine
Baltimore, Maryland, United States, 21287
Actively Recruiting
4
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
6
New York University
New York, New York, United States, 10016
Actively Recruiting
7
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
8
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
9
Hopital Louis Pradel
Bron, France
Actively Recruiting
10
Nantes University Hospital
Nantes, France, 44000
Actively Recruiting
11
Pitié-Salpêtrière Hospital
Paris, France, 75013
Actively Recruiting
12
Hôpital Haut-Lévêque
Pessac, France, 33604
Actively Recruiting
13
University Hospital Muenster
Münster, Germany, 48149
Actively Recruiting
14
Wuerzburg University Hospital
Würzburg, Germany, 97080
Actively Recruiting
15
Centro Cardiologico Monzino
Milan, Italy, 20138
Actively Recruiting
16
Istituti Clinici Scientifici Maugeri SpA
Pavia, Italy
Actively Recruiting
17
Skåne University Hospital
Malmö, Sweden, 214 28
Actively Recruiting
18
The Queen Elizabeth Hospital
Glasgow, United Kingdom, G514TF
Actively Recruiting
19
Barts & The London Health NHS Trust
London, United Kingdom, E1 1BB
Actively Recruiting
20
St. George's University Hospitals NHS Foundation Trust
London, United Kingdom, SW17 0QT
Actively Recruiting
21
Royal Brompton & Harefield NHS Foundation Trust
London, United Kingdom, SW3 6NP
Actively Recruiting
Research Team
M
Matthew Pollman, MD
N
Niharika Kamat, MS
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here