Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
NCT ID: NCT06228924
Last Updated: 2025-02-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
15 participants
INTERVENTIONAL
2024-03-26
2029-10-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Cohort 1
Dose for Cohort 1 will be 3E13 vg/kg
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.
Cohort 2
Dose for Cohort 2 will be 6E13 vg/kg
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.
Interventions
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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.
Eligibility Criteria
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Inclusion Criteria
* 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)
Exclusion Criteria
* High AAV9 neutralizing antibody titer
* Prior myocardial infarction
* Right Ventricular Heart Failure
* Class IV Heart Failure
* Clinically significant renal disease
* Clinically significant liver disease
18 Years
65 Years
ALL
No
Sponsors
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Tenaya Therapeutics
INDUSTRY
Responsible Party
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Locations
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University of California San Francisco
San Francisco, California, United States
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, United States
Johns Hopkins University
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
NYU Langone Health
New York, New York, United States
Cleveland Clinic
Cleveland, Ohio, United States
Countries
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Central Contacts
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Facility Contacts
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Vasanth Vedantham, MD
Role: primary
Matthew Taylor, MD
Role: primary
Neal Lakdawala, MD
Role: primary
Larry Chinitz, MD
Role: primary
Milind Desai, MD
Role: primary
Related Links
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Other Identifiers
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TN-401-0012
Identifier Type: -
Identifier Source: org_study_id
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