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Taking people in FranceNo phase

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.

Key facts

Who runs it
Tenaya Therapeutics
Where
Bron, Nantes, Paris, and Pessac, FranceSee the 4 sitesAlso in 5 other countries, 17 sites
Trial code
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)
Status
Recruiting
People taking part
200 people, planned
Started
31 Jan 2023
Expected to end
11 Jul 2030
Type of study
Observational
Record updated
1 November 2024

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for people aged 14 to 65 with arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by a PKP2 gene change. It follows how the disease progresses and checks for antibodies against AAV9, a virus vector used in gene therapy.

Age
14 to 65 years
Sex
All sexes can join.
Disease stage
ARVC diagnosed and meeting the 2010 Modified Task Force Criteria for ARVC as affected. A working ICD (implanted heart device) is required.
Earlier treatment
People who ever received any investigational or approved gene therapy cannot join. People currently on systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy cannot join.
Biomarker or test result
A pathogenic or likely pathogenic PKP2 gene mutation.

Main reasons for not taking part

  • Currently on immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
  • Clinically significant liver disease, hepatitis B, hepatitis C, HIV or tuberculosis infection
  • Earlier gene therapy of any kind
  • Taking part in another interventional trial, unless the Sponsor approves
  • History of heart transplant

The registry criteria, word for word

Show all 9 criteriaShow less

Who can take part

  • 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 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

Check if you might fit

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?

Where in France

Also in 5 other countries, 17 sites

  • Hôpital Louis PradelBron, FranceContact: Philippe Chevalier, MD, PhDTaking people
  • Nantes University HospitalNantes, FranceContact: Vincent Probst, MDTaking people
  • Pitié Salpêtrière HospitalParis, FranceContact: Estelle Gandjbakhch, MD, PhDTaking people
  • Hopital Haut-LevequePessac, FranceContact: Frederic Sacher, MD, PhDTaking people

About the trial

What the trial wants to find out

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.

The group in the trial

  • Retrospective and Prospective

    Patients who meet the eligibility criteria are observed and data collected both prospectively and retrospectively.

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Plain-language title
Studies antibodies to a gene therapy carrier in people with PKP2 heart diseaseGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Fit questions
Built from NCT06311708, who can take part
Record updated
1 November 2024