Recruiting

Observational Study

Sponsor:

Tenaya Therapeutics

Code:

NCT06311708

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 14 - 65

Healthy Volunteers: Not accepted

Study Details

Brief 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.

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Study ID

NCT06311708

Start date

Jan 31, 2023

Status verified date

Oct, 2024

Completion date

Jul 11, 2030

Anticipated

Primary completion date

Mar 29, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 14 - 65

Healthy Volunteers: Not accepted

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

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Arms

Retrospective and Prospective

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

Primary outcome measure

  • Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Matthew Pollman, MD

650-209-8092mpollman@tenayathera.com

Niharika Kamat, MS

clinical.trials@tenayathera.com

Locations

University of California San Francisco

Recruiting

San Francisco, California, United States, 94143

University of Colorado, Denver

Recruiting

Aurora, Colorado, United States, 80045

John Hopkins University School of Medicine

Recruiting

Baltimore, Maryland, United States, 21287

Brigham and Women's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

New York University

Recruiting

New York, New York, United States, 10016

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Medical University of South Carolina

Recruiting

Charleston, South Carolina, United States, 29425

More Information

Sponsor

Tenaya Therapeutics

Last update posted

Nov 1, 2024

Last verified

Oct, 2024

Keywords

  • PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
  • Arrhythmogenic Cardiomyopathy (ACM)
  • PKP2-associated ARVC
  • PKP2-ARVC
  • PKP2-ACM
  • Adeno Associated Virus (AAV)
  • Adeno-Associated Virus Serotype 9 (AAV9)
  • Gene Therapy
  • Gene Transfer
  • Genetic cardiomyopathy
  • Heart Failure

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Tenaya Therapeutics on 2024-11-01.