NCT06228924

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
41mo left

Started Mar 2024

Longer than P75 for phase_1

Geographic Reach
1 country

7 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress38%
Mar 2024Oct 2029

First Submitted

Initial submission to the registry

January 18, 2024

Completed
11 days until next milestone

First Posted

Study publicly available on registry

January 29, 2024

Completed
2 months until next milestone

Study Start

First participant enrolled

March 26, 2024

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2026

Completed
3.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2029

Expected
Last Updated

February 6, 2025

Status Verified

September 1, 2024

Enrollment Period

2.1 years

First QC Date

January 18, 2024

Last Update Submit

February 5, 2025

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Keywords

PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)Arrhythmogenic Cardiomyopathy (ACM)PKP2-associated ARVCPKP2-ARVCPKP2-ACMAdeno Associated Virus (AAV)Gene TherapyGene TransferGenetic cardiomyopathyHeart Failure

Outcome Measures

Primary Outcomes (2)

  • Number and severity of Adverse Events over the course of the study.

    52 weeks

  • Number of Serious Adverse Events related to study drug.

    5 years

Secondary Outcomes (3)

  • To assess changes in daily PVC and NSVT counts

    Week 52

  • To assess frequency of ICD therapy administration

    Week 52

  • To assess frequency of sustained VT

    Week 52

Study Arms (2)

Cohort 1

EXPERIMENTAL

Dose for Cohort 1 will be 3E13 vg/kg

Genetic: TN-401

Cohort 2

EXPERIMENTAL

Dose for Cohort 2 will be 6E13 vg/kg

Genetic: TN-401

Interventions

TN-401GENETIC

TN-401 is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy containing Plakophilin-2 (PKP2) transgene. It is a single (one-time) intravenous dose.

Cohort 1Cohort 2

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 may not qualify 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

University of California San Francisco

San Francisco, California, 94143, United States

RECRUITING

University of Colorado - Anschutz Medical Campus

Aurora, Colorado, 80045, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

NYU Langone Health

New York, New York, 10016, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Arrhythmogenic Right Ventricular DysplasiaHeart Failure

Condition Hierarchy (Ancestors)

Heart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesCardiomyopathiesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Matthew Pollman, M.D.

CONTACT

(650) 416-1186mpollman@tenayathera.com

Niha Kamat

CONTACT

clinical.trials@tenayathera.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 18, 2024

First Posted

January 29, 2024

Study Start

March 26, 2024

Primary Completion

May 1, 2026

Study Completion (Estimated)

October 1, 2029

Last Updated

February 6, 2025

Record last verified: 2024-09

Data Sharing

IPD Sharing
Will not share

Locations

US(7)