Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorTenaya Therapeutics
About this trial
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.
Eligibility criteria
Qualifiers
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 at least 9 months prior to Screening
Disqualifiers
Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening
High AAV9 neutralizing antibody titer
Prior myocardial infarction
Right Ventricular Heart Failure
Trial design
Treatments tested in this trial
- TN-401
Treatment groups
15 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov