Studies antibodies to a gene therapy carrier in people with PKP2 heart disease
Observes disease course and checks antibodies to a gene therapy carrier in patients.
Plain-language title generated from the registry record
Registry 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)
Key facts
- Where
- Münster and Würzburg, GermanySee the 2 sites
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 14 and 65 years old?
Source: NCT06311708, who can take part
Where in Germany
- University hospital MuensterMünster, GermanyTaking people
- Wuerzburg University HospitalWürzburg, GermanyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Tenaya Therapeutics
- Trial code
- NCT06311708
- Record updated
- 1 November 2024
- Original description
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.Read full original descriptionShow less
Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
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.
Inclusion: 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: Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy; 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 clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection; History of cardiac transplant.