ARVC heart disease
Conditions
Interventions
None listed
Sponsors
Tenaya Therapeutics Inc.
Eligibility
Age
12 Years to 64 Years
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| AAV antibody titers | — |
Secondary
| Measure | Time frame |
|---|---|
| • Incidence of cardiac hospitalizations, cardiac events, including arrhythmias and ICD therapy administration, cardiac-related procedures, including catheter-based ablations and treatments, cardiac transplantation, and death • Serial Holter (or equivalent) monitor examinations • New York Heart Association (NYHA) functional classification • Activity restrictions • QoL questionnaires • ECHO parameters and other available imaging modalities, as per local standard of care, in terms of heart structure and function | — |
Countries
Netherlands
Outcome results
None listed