NCT05836259 · Tenaya Therapeutics
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
(MyPEAK-1)
What this study is about
This is a first-in-human, non-randomly assigned, where both patients and doctors know the treatment given study designed to evaluate the safety, tolerability, and how the drug affects the body (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.
View original scientific description
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.
Interventions
GENETIC
TN-201
TN-201 is a recombinant adeno-associated virus serotype 9 (AAV9) containing Myosin Binding Protein C (MYBPC3) transgene. It is a single (one-time) intravenous dose.
Primary outcome measures
Number and severity of Adverse Events over the course of the study.
Time frame: 5 Years
Number of Serious Adverse Events related to study drug.
Time frame: 5 Years
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- MYBPC3 mutation
- Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
- Left Ventricular Ejection Fraction ≥45%
- NYHA Functional Class II or III symptoms
- NT-proBNP ≥160pg/ml
Exclusion criteria
- High AAV9 neutralizing antibody titer
Where
- La Jolla, California
- San Francisco, California
- Atlanta, Georgia
- Boston, Massachusetts
- Rochester, Minnesota
- Cincinnati, Ohio
- Cleveland, Ohio
- Portland, Oregon
- Philadelphia, Pennsylvania
- Houston, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 19, 2026 · Source of record for eligibility and locations