At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 90 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
In plain English
This is a non-interventional (observational) study, which means it does not give you any treatment. It looks at whether patients with a heart condition called arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by a change in the PKP2 gene already have antibodies (protective proteins made by the immune system) against a virus carrier called AAV9, and how their disease changes over time. The study is run by Tenaya Therapeutics.
Who can take part
- Aged 14 to 65 when agreeing to take part
- Have a harmful or likely harmful change (mutation) in the PKP2 gene
- Have been diagnosed with ARVC and meet the 2010 Modified Task Force Criteria as affected
- Have a working ICD (a device placed in the chest that controls dangerous heart rhythms)
Who may not be able to
- Currently taking medicines that weaken the immune system, certain chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy
- Have had serious liver disease, hepatitis B, hepatitis C, HIV, or tuberculosis
- Have ever received any gene therapy, whether experimental or approved
- Are taking part in another interventional (treatment) trial, unless the sponsor approves it (joining another observational study may be allowed)
- Have had a heart transplant
What taking part involves
- • This study does not involve any treatment — it only collects information. What will be done during the study is not stated — ask the trial team.
Time commitment: Number of visits, how long the study lasts, and what taking part involves are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- 14 Years to 65 Years
- Who
- All
- Number of participants
- 200
- Started
- 2023-01-31
- Last checked
- 2024-10
Plain English Summary
What is this study?
- • Testing a new treatment for arrhythmogenic right ventricular cardiomyopathy
- • Clinical study - 200 participants
- • 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
Who can take part?
- • Ages 14 Years to 65 Years
- • Diagnosed with arrhythmogenic right ventricular cardiomyopathy
Where?
- • Glasgow - The Queen Elizabeth Hospital
- • London - Barts & The London Health NHS Trust
- • London - St. George's University Hospitals NHS Foundation Trust
- • London - Royal Brompton & Harefield NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
Patients will receive standard of care treatments and assessments under the care of their healthcare provider. Biologic samples will be collected annually to measure cardiac and other related biomarkers. 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. If consent is provided, there may be a one-time sample collection to evaluate genetics for research purposes. Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life. If not included as part of a patient's standard of care, diagnostic Holter (or equivalent) monitoring will be required annually. No investigational product will be administered. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 14 Years - 65 Years
- Who can join: All genders
What the study is looking for
- ✓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
Who cannot take part
- ✗Currently receiving systemic immunosuppressive therapy, cytotoxic drug treatment, immunoglobulin therapy or monoclonal...
- ✗History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus,...
- ✗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...
- ✗History of heart transplant.
See the full criteria
Where Is This Study? (4 UK sites)
The Queen Elizabeth Hospital
Glasgow G514TF, United Kingdom
Barts & The London Health NHS Trust
London E1 1BB, United Kingdom
St. George's University Hospitals NHS Foundation Trust
London SW17 0QT, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust
London SW3 6NP, United Kingdom
How to Get in Touch
Matthew Pollman, MD
Sponsor contactCONTACT
Niharika Kamat, MS
Sponsor contactCONTACT