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

Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.

Participants needed: 200

Trial details

Age: 0-18Biological sex: AllType: ObservationalSponsor: Tenaya TherapeuticsUpdated: Nov 13, 2024Locations: 29

Eligibility criteria

Data is available for patient <18 years of age. Patients must be <18 years of ag... [+1]

Patient received cardiac transplantation or died >10 years before study initiati... [+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

Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

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)