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

Part of paid clinical trials in San Francisco, California.

Sponsor: Tenaya Therapeutics
Study ID: NCT06228924
Phase: PHASE1
Status: Recruiting

Apply to this trial

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Eligibility Criteria

Sex: ALL
Age: 18 Years - 65 Years
Healthy Volunteers: Not accepted

Interventions

TN-401 — GENETIC
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.

Study Details

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.

Key Dates

Start date: Mar 26, 2024
Status verified: Sep 2024
Primary completion: May 1, 2026
Completion: Oct 1, 2029

Study Design

Enrollment: 15 participants (estimated)
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Arms

Experimental: Cohort 1
Dose for Cohort 1 will be 3E13 vg/kg
Experimental: Cohort 2
Dose for Cohort 2 will be 6E13 vg/kg

Primary Outcome Measure

Number and severity of Adverse Events over the course of the study. [ Time Frame: 52 weeks ]

Central Contacts

Matthew Pollman, M.D.
(650) 416-1186
[email protected]
Niha Kamat
[email protected]

Locations (7)

Facility	City	State	ZIP	Site coordinators
University of California San Francisco	San Francisco	California	94143	Vasanth Vedantham, MD
University of Colorado - Anschutz Medical Campus	Aurora	Colorado	80045	Matthew Taylor, MD
Johns Hopkins University	Baltimore	Maryland	21287	Andreas Barth, MD (PRINCIPAL_INVESTIGATOR)
Brigham and Women's Hospital	Boston	Massachusetts	02115	Neal Lakdawala, MD
Mayo Clinic	Rochester	Minnesota	55905	John R. Giudicessi, MD (PRINCIPAL_INVESTIGATOR)
NYU Langone Health	New York	New York	10016	Larry Chinitz, MD
Cleveland Clinic	Cleveland	Ohio	44195	Milind Desai, MD

Find similar trials in San Francisco, CA

By research site

University of California San Francisco· San Francisco, CA University of Colorado - Anschutz Medical Campus· Aurora, CO Johns Hopkins University· Baltimore, MD Brigham and Women's Hospital· Boston, MA Mayo Clinic· Rochester, MN NYU Langone Health· New York, NY

Related Studies

Mayo AVC Registry and Biobank
Recruiting · Mayo Clinic · Rochester, Minnesota
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)
Recruiting · Tenaya Therapeutics · San Francisco, California
Pediatric Cardiomyopathy Mutation Analysis
Recruiting · Indiana University · Indianapolis, Indiana