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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06311708
Other study ID # TN-401-0011
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date January 31, 2023
Est. completion date July 11, 2030

Study information

Verified date May 2024
Source Tenaya Therapeutics
Contact Matthew Pollman, MD
Phone 6504161186
Email mpollman@tenayathera.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Description:

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date July 11, 2030
Est. primary completion date March 29, 2030
Accepts healthy volunteers No
Gender All
Age group 14 Years to 65 Years
Eligibility Inclusion Criteria: - 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 Criteria: - Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy - History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection - 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 cardiac transplant.

Study Design


Related Conditions & MeSH terms

  • Arrhythmogenic Right Ventricular Cardiomyopathy
  • Arrhythmogenic Right Ventricular Dysplasia
  • Cardiomyopathies

Locations

Country Name City State
France Hopital Louis Pradel Bron
France Nantes University Hospital Nantes
France Pitié-Salpêtrière Hospital Paris
France Hôpital Haut-Lévêque Pessac
Germany Ludwig-Maximilians - University of Munich München
Germany University Hospital Muenster Münster
Germany Wuerzburg University Hospital Würzburg
Italy Centro Cardiologico Monzino Milano
Italy Istituti Clinici Scientifici Maugeri SpA Pavia
Sweden University Hospital, Linkoeping Linköping
Sweden Skåne University Hospital Malmö
United Kingdom The Queen Elizabeth Hospital Glasgow
United Kingdom Barts & The London Health NHS Trust London
United Kingdom Royal Brompton & Harefield NHS Foundation Trust London
United Kingdom St. George's University Hospitals NHS Foundation Trust London
United States University of Colorado, Denver Aurora Colorado
United States John Hopkins University School of Medicine Baltimore Maryland
United States Brigham and Women's Hospital Boston Massachusetts
United States Medical University of South Carolina Charleston South Carolina
United States Cleveland Clinic Cleveland Ohio
United States New York University New York New York
United States Mayo Clinic Rochester Minnesota
United States University of California San Francisco San Francisco California

Sponsors (24)

Lead Sponsor Collaborator
Tenaya Therapeutics Barts & The London NHS Trust, Brigham and Women's Hospital, Centro Cardiologico Monzino, Hôpital Haut-Lévêque, Hopital Louis Pradel, Istituti Clinici Scientifici Maugeri SpA, Johns Hopkins University, Ludwig-Maximilians - University of Munich, Mayo Clinic, Medical University of South Carolina, Nantes University Hospital, New York University, Pitié-Salpêtrière Hospital, Royal Brompton & Harefield NHS Foundation Trust, Skane University Hospital, St George's University Hospitals NHS Foundation Trust, The Cleveland Clinic, The Queen Elizabeth Hospital, University Hospital Muenster, University Hospital, Linkoeping, University of California, San Francisco, University of Colorado, Denver, Wuerzburg University Hospital

Countries where clinical trial is conducted

United States,  France,  Germany,  Italy,  Sweden,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Other • To monitor biomarkers associated with disease progression and inflammation in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
Other To evaluate genetics associated with PKP2-associated ARVC 5 years
Other To evaluate the effect of other cardiac mutations or other genetic variants that might affect the penetrance and/or expressivity of PKP2 mutations in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
Other To evaluate health care utilization in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
Primary Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC 5 years
Secondary To characterize the burden of illness in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
Secondary To characterize arrhythmic risk in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
Secondary To evaluate functional status and Quality of Life (QoL) in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
Secondary To evaluate heart function as assessed by imaging in patients with pathogenic or likely pathogenic PKP2 mutations 5 years
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