Status: Recruiting

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

Participants needed: 30

Trial details

Phase: Phase 1, Phase 2Age: 18-75Biological sex: AllType: InterventionalSponsor: Tenaya TherapeuticsUpdated: Mar 19, 2026Locations: 10

Eligibility criteria

MYBPC3 mutation [+4]

High AAV9 neutralizing antibody titer

Status: Recruiting

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

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.

Participants needed: 15

Trial details

Phase: Phase 1Age: 18-65Biological sex: AllType: InterventionalSponsor: Tenaya TherapeuticsUpdated: Feb 6, 2025Locations: 7

Eligibility criteria

PKP2 mutation (pathogenic or likely pathogenic) [+5]

Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT... [+6]

Status: Recruiting

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)

This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

Participants needed: 200

Trial details

Age: 14-65Biological sex: AllType: ObservationalSponsor: Tenaya TherapeuticsUpdated: Nov 1, 2024Locations: 21

Eligibility criteria

Ages 14-65 years, inclusive, at the time of consent [+3]

Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy,... [+4]

Clinical trials

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

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

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)