Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV) in Patients with Plakophilin 2 (PKP2)-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
- Conditions
- ARVCheart disease10028593
- Registration Number
- NL-OMON56804
- Lead Sponsor
- Tenaya Therapeutics Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 30
1. Ages 14 to 65 years, inclusive, at time of consent
2. Pathogenic or likely pathogenic PKP2 gene mutation
3. Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC
4. Functioning ICD
1. Currently receiving systemic immunosuppressive therapy, cytotoxic
chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy
2. History of clinically significant liver disease, hepatitis B virus,
hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
3. Previously dosed with any investigational or approved gene therapy product
at any time
4. 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
5. History of cardiac transplant
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>AAV antibody titers</p><br>
- Secondary Outcome Measures
Name Time Method <p>• Incidence of cardiac hospitalizations, cardiac events, including arrhythmias<br /><br>and ICD therapy administration, cardiac-related procedures, including<br /><br>catheter-based ablations and treatments, cardiac transplantation, and death<br /><br><br /><br>• Serial Holter (or equivalent) monitor examinations<br /><br><br /><br>• New York Heart Association (NYHA) functional classification<br /><br>• Activity restrictions<br /><br>• QoL questionnaires<br /><br><br /><br>• ECHO parameters and other available imaging modalities, as per local standard<br /><br>of care, in terms of heart structure and function</p><br>
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