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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
15
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.
University of California San Francisco
San Francisco, California, United States
RECRUITINGUniversity of Colorado - Anschutz Medical Campus
Aurora, Colorado, United States
RECRUITINGJohns Hopkins University
Baltimore, Maryland, United States
Number and severity of Adverse Events over the course of the study.
Time frame: 52 weeks
Number of Serious Adverse Events related to study drug.
Time frame: 5 years
To assess changes in daily PVC and NSVT counts
Time frame: Week 52
To assess frequency of ICD therapy administration
Time frame: Week 52
To assess frequency of sustained VT
Time frame: Week 52
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Brigham and Women's Hospital
Boston, Massachusetts, United States
RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITINGNYU Langone Health
New York, New York, United States
RECRUITINGCleveland Clinic
Cleveland, Ohio, United States
RECRUITING