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)
Status: Recruiting
Location: See all (10) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:
• MYBPC3 mutation
• Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
• Left Ventricular Ejection Fraction ≥45%
• NYHA Functional Class II or III symptoms
• NT-proBNP ≥160pg/ml
Locations
United States
California
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
RECRUITING
La Jolla
University of California San Francisco
RECRUITING
San Francisco
Georgia
Emory University
WITHDRAWN
Atlanta
Massachusetts
Brigham and Women's Hospital
WITHDRAWN
Boston
Minnesota
Mayo Clinic
RECRUITING
Rochester
Ohio
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
WITHDRAWN
Cincinnati
Cleveland Clinic
RECRUITING
Cleveland
Oregon
Oregon Health & Science University
WITHDRAWN
Portland
Pennsylvania
Hospital of the University of Pennsylvania
WITHDRAWN
Philadelphia
Texas
Houston Methodist Hospital
RECRUITING
Houston
Contact Information
Primary
Matt Pollman, M.D.
mpollman@tenayathera.com
650-209-8092
Backup
LaTanya Tomlinson, RN, MHSA
clinical.trials@tenayathera.com
650-825-6990
Time Frame
Start Date: 2023-08-10
Estimated Completion Date: 2032-08
Participants
Target number of participants: 30
Treatments
Experimental: Cohort 1
Dose for Cohort 1 will be 3E13 vg/kg
Experimental: Cohort 2
Dose for Cohort 2 will be 6E13 vg/kg
Related Therapeutic Areas
Sponsors
Leads: Tenaya Therapeutics