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

NCT06228924 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-02-06

No results posted yet for this study

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.

Conditions

  • Arrhythmogenic Right Ventricular Cardiomyopathy

Interventions

GENETIC

TN-401

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.

Sponsors & Collaborators

  • Tenaya Therapeutics

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-26
Primary Completion
2026-05-01
Completion
2029-10-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06228924 on ClinicalTrials.gov