Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC

a study on Cardiomyopathy Dysplasia

Summary

Eligibility
for people ages 18-65 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

Summary

This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.

Official Title

First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-401, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, in Adults With PKP2 Mutation-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

Details

The RIDGE-1™ open-label Safety, Tolerability, Dose-finding, PD and Cardiac Transgene Expression Study will enroll up to 15 patients in two planned dose cohorts. Patients in each cohort will receive a single intravenous (IV) dose of TN-401. Following Data Safety Monitoring Board (DSMB) review of each dose cohort, the next dose cohort will be initiated. DSMB review will also be needed to expand the dose cohorts. The dose for Cohorts 1/1a will be 3E13 (3 × 1013) vg/kg and the dose for Cohorts 2/2a will be 6E13 (6 × 1013) vg/kg.

Keywords

Arrhythmogenic Right Ventricular Cardiomyopathy, PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), Arrhythmogenic Cardiomyopathy (ACM), PKP2-associated ARVC, PKP2-ARVC, PKP2-ACM, Adeno Associated Virus (AAV), Gene Therapy, Gene Transfer, Genetic cardiomyopathy, Heart Failure, Cardiomyopathies, Arrhythmogenic Right Ventricular Dysplasia

Eligibility

You can join if…

Open to people ages 18-65

  • PKP2 mutation (pathogenic or likely pathogenic)
  • Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria
  • Left Ventricular Ejection Fraction ≥50%
  • Functioning Implantable Cardiac Defibrillator with remote integration capabilities at least 9 months prior to Screening
  • NYHA Functional Class I, II, or III
  • Frequent premature ventricular contractions (PVCs)

You CAN'T join if...

  • Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening
  • High AAV9 neutralizing antibody titer
  • Prior myocardial infarction
  • Right Ventricular Heart Failure
  • Class IV Heart Failure
  • Clinically significant renal disease
  • Clinically significant liver disease

Locations

  • UCSF accepting new patients
    San Francisco California 94143 United States
  • University of Colorado - Anschutz Medical Campus accepting new patients
    Aurora Colorado 80045 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Tenaya Therapeutics
Links
Related Info Related Info #2 Related Info #3 Related Info #4
ID
NCT06228924
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 15 study participants
Last Updated
Contact us about this trial