Actively Recruiting

Age: 14Years - 65Years
All Genders
NCT06311708

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)

Led by Tenaya Therapeutics · Updated on 2024-11-01

200

Participants Needed

21

Research Sites

388 weeks

Total Duration

On this page

Sponsors

T

Tenaya Therapeutics

Lead Sponsor

U

University of California, San Francisco

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official 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)

Who Can Participate

Age: 14Years - 65Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Ages 14 to 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 implantable cardioverter defibrillator (ICD)
Not Eligible

You will not qualify if you...

  • 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
  • Concurrent participation in another interventional clinical trial unless approved by the Sponsor
  • History of cardiac transplant

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 21 locations

1

University of California San Francisco

San Francisco, California, United States, 94143

Actively Recruiting

2

University of Colorado, Denver

Aurora, Colorado, United States, 80045

Actively Recruiting

3

John Hopkins University School of Medicine

Baltimore, Maryland, United States, 21287

Actively Recruiting

4

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

Actively Recruiting

5

Mayo Clinic

Rochester, Minnesota, United States, 55905

Actively Recruiting

6

New York University

New York, New York, United States, 10016

Actively Recruiting

7

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Actively Recruiting

8

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

Actively Recruiting

9

Hopital Louis Pradel

Bron, France

Actively Recruiting

10

Nantes University Hospital

Nantes, France, 44000

Actively Recruiting

11

Pitié-Salpêtrière Hospital

Paris, France, 75013

Actively Recruiting

12

Hôpital Haut-Lévêque

Pessac, France, 33604

Actively Recruiting

13

University Hospital Muenster

Münster, Germany, 48149

Actively Recruiting

14

Wuerzburg University Hospital

Würzburg, Germany, 97080

Actively Recruiting

15

Centro Cardiologico Monzino

Milan, Italy, 20138

Actively Recruiting

16

Istituti Clinici Scientifici Maugeri SpA

Pavia, Italy

Actively Recruiting

17

Skåne University Hospital

Malmö, Sweden, 214 28

Actively Recruiting

18

The Queen Elizabeth Hospital

Glasgow, United Kingdom, G514TF

Actively Recruiting

19

Barts & The London Health NHS Trust

London, United Kingdom, E1 1BB

Actively Recruiting

20

St. George's University Hospitals NHS Foundation Trust

London, United Kingdom, SW17 0QT

Actively Recruiting

21

Royal Brompton & Harefield NHS Foundation Trust

London, United Kingdom, SW3 6NP

Actively Recruiting

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Research Team

M

Matthew Pollman, MD

CONTACT

N

Niharika Kamat, MS

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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