Hypertrophic CardiomyopathyPhase 1/2MyPEAK-1NCT05836259
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
A Phase 1/2 study testing TN-201 in people with Hypertrophic Cardiomyopathy, run by Tenaya Therapeutics at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Hypertrophic Cardiomyopathy
- What is being tested?
- TN-201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 10 sitesUnited States
- Study dates
- 10 Aug 2023to Aug 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- MYBPC3 mutation
- Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
- Left Ventricular Ejection Fraction ≥45%
- NYHA Functional Class II or III symptoms
- NT-proBNP ≥160pg/ml
Who cannot join
- High AAV9 neutralizing antibody titer
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
United States10 sites, 5 recruiting
- Withdrawn
Emory University
Atlanta, Georgia, 30322
- Withdrawn
Brigham and Women's Hospital
Boston, Massachusetts, 02115
- Withdrawn
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
Cincinnati, Ohio, 45219
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
Rachael Landis, 216-444-3650, LANDISR@ccf.org
- Recruiting
Houston Methodist Hospital
Houston, Texas, 77030
Jennifer Garrett, 281-222-9983, jmgarrett@houstonmethodist.org
- Recruiting
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
La Jolla, California, 92093
Matt Fahey, 858-534-3798, mjfahey@health.ucsd.edu
- Withdrawn
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
- Withdrawn
Oregon Health & Science University
Portland, Oregon, 97239
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Sierra Slade, 507-422-5433, Slade.Sierra@mayo.edu
- Recruiting
University of California San Francisco
San Francisco, California, 94117
Emma Reagan, 415-514-7280, Emma.reagan@ucsf.edu
About this study
Official title: 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)
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.
Read the detailed description
The study will consist of 2 escalating dose cohorts (groups). The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug (TN-201 Gene Therapy). The study will follow patients for 5 years following a single dose of TN-201.
- Conditions
- Hypertrophic Cardiomyopathy
- Being tested
- TN-201
- Keywords
- Hypertrophic Cardiomyopathy (HCM), Myosin Binding Protein C3 (MYBPC3), Nonobstructive HCM, Genetic HCM, Familial HCM, Adenoassociated Virus (AAV), Gene Therapy, Obstructive HCM, nHCM, oHCM
- Medical subject headings
- Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TN-201Genetic
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Dose for Cohort 1 will be 3E13 vg/kg Receives: Genetic: TN-201 |
| Cohort 2 | Experimental | Dose for Cohort 2 will be 6E13 vg/kg Receives: Genetic: TN-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and severity of Adverse Events over the course of the study.
Measured: 5 Years
Number of Serious Adverse Events related to study drug.
Measured: 5 Years
Secondary outcomes (1)
Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).
The score ranges from 0-100, with zero being a worse outcome
Measured: 52 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2023
- Study start10 Aug 2023
- Primary completionAug 2027 (estimated)
- Study completionAug 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- This is a first-in-human, non-randomized, open label Phase 1b/2 study. The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug.
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tenaya Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05836259
- Sponsor's protocol ID
- TN-201-0009
- Acronym
- MyPEAK-1
- Status last verified
- Mar 2026
- First submitted
- 18 Apr 2023
- Last updated
- 19 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Grisorio L, Bongianino R, Gianeselli M, Priori SG. Gene therapy for cardiac diseases: methods, challenges, and future directions. Cardiovasc Res. 2024 Nov 25;120(14):1664-1682. doi: 10.1093/cvr/cvae207.
PubMed 39302117
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT05836259
- Acronym / sponsor ID
- MyPEAK-1 / TN-201-0009
- Conditions
- Hypertrophic Cardiomyopathy
- Keywords
- Hypertrophic Cardiomyopathy (HCM), Myosin Binding Protein C3 (MYBPC3), Nonobstructive HCM, Genetic HCM, Familial HCM, Adenoassociated Virus (AAV), Gene Therapy, Obstructive HCM
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Tenaya TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 10 Aug 2023 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Aug 2032 (estimated)
- First posted
- 1 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The study will consist of 2 escalating dose cohorts (groups). The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug (TN-201 Gene Therapy). The study will follow patients for 5 years following a single dose of TN-201.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This is a first-in-human, non-randomized, open label Phase 1b/2 study. The study will consist of 2 escalating dose cohorts. The study will enroll at least 6 and as many as 30 patients. All patients will receive active drug.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: TN-201Dose for Cohort 1 will be 3E13 vg/kg |
| Cohort 2 | Experimental | Genetic: TN-201Dose for Cohort 2 will be 6E13 vg/kg |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- MYBPC3 mutation
- Hypertrophic Cardiomyopathy (obstructive and nonobstructive)
- Left Ventricular Ejection Fraction ≥45%
- NYHA Functional Class II or III symptoms
- NT-proBNP ≥160pg/ml
Key exclusion criteria (1 listed)
- High AAV9 neutralizing antibody titer
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TN-201TN-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. | Genetic | Cohort 1, Cohort 2 |
Primary outcome
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
1 secondary outcome
Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).
Time frame: 52 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2026.
- Derived from this study: PubMed 39302117Grisorio L, Bongianino R, Gianeselli M, Priori SG. Gene therapy for cardiac diseases: methods, challenges, and future directions. Cardiovasc Res. 2024 Nov 25;120(14):1664-1682. doi: 10.1093/cvr/cvae207.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TN-201 in people with Hypertrophic Cardiomyopathy, run by Tenaya Therapeutics at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Matt Pollman, M.D. (650-209-8092, mpollman@tenayathera.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number and severity of Adverse Events over the course of the study. (time frame: 5 Years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05836259, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2026.