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Trial record 1 of 2 for:    Tenaya Therapeutics | ARVC
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Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (RIDGE-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT06228924
Recruitment Status : Recruiting
First Posted : January 29, 2024
Last Update Posted : March 29, 2024
Sponsor:
Collaborators:
Johns Hopkins University
Mayo Clinic
Information provided by (Responsible Party):
Tenaya Therapeutics

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.

Condition or disease Intervention/treatment Phase
Arrhythmogenic Right Ventricular Cardiomyopathy Genetic: TN-401 Phase 1

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Estimated Study Start Date : April 1, 2024
Estimated Primary Completion Date : May 1, 2026
Estimated Study Completion Date : October 1, 2029


Arm Intervention/treatment
Experimental: Cohort 1
Dose for Cohort 1 will be 3E13 vg/kg
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.

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




Primary Outcome Measures :
  1. Number and severity of Adverse Events over the course of the study. [ Time Frame: 52 weeks ]
  2. Number of Serious Adverse Events related to study drug. [ Time Frame: 5 years ]

Secondary Outcome Measures :
  1. To assess changes in daily PVC and NSVT counts [ Time Frame: Week 52 ]
  2. To assess frequency of ICD therapy administration [ Time Frame: Week 52 ]
  3. To assess frequency of sustained VT [ Time Frame: Week 52 ]


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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 ≥12 months prior to enrollment
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06228924


Contacts
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Contact: Matthew Pollman, M.D. (650) 416-1186 mpollman@tenayathera.com
Contact: Niha Kamat clinical.trials@tenayathera.com

Locations
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United States, Maryland
Johns Hopkins University Not yet recruiting
Baltimore, Maryland, United States, 21287
Principal Investigator: Andreas Barth, MD         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Principal Investigator: John R. Giudicessi, MD         
Sponsors and Collaborators
Tenaya Therapeutics
Johns Hopkins University
Mayo Clinic
Additional Information:
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Responsible Party: Tenaya Therapeutics
ClinicalTrials.gov Identifier: NCT06228924    
Other Study ID Numbers: TN-401-0012
First Posted: January 29, 2024    Key Record Dates
Last Update Posted: March 29, 2024
Last Verified: March 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Tenaya Therapeutics:
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
Additional relevant MeSH terms:
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Cardiomyopathies
Arrhythmogenic Right Ventricular Dysplasia
Heart Diseases
Cardiovascular Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Congenital Abnormalities