First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-201, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, in Adults With MYBPC3 Mutation-associated Hypertrophic Cardiomyopathy (HCM) (MyPEAK-1)

NCT05836259 Phase 1/Phase 2 RECRUITING

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.

Details

Lead sponsor	Tenaya Therapeutics
Phase	Phase 1/Phase 2
Status	RECRUITING
Enrolment	30
Start date	2023-08-10
Completion	2032-08

Conditions

• Hypertrophic Cardiomyopathy

Interventions

• TN-201

Primary outcomes

• Number and severity of Adverse Events over the course of the study. — 5 Years
• Number of Serious Adverse Events related to study drug. — 5 Years

Countries

United States