MyPEAK-1:
A Clinical Trial for People with MYBPC3-associated Hypertrophic Cardiomyopathy (HCM)
The MyPEAK-1 clinical trial will research an investigational gene therapy called TN-201 for the treatment of HCM caused by mutations (changes) in the MYBPC3 gene. Changes in the MYBPC3 gene prevent certain cells from making a protein needed for the heart to pump as expected.
TN-201 is an investigational gene therapy designed to address the underlying genetic cause of HCM.
- TN-201 uses an AAV9 capsid to deliver a working MYBPC3 gene to the muscle cells in the heart
- Once in these cells, the working gene delivered by TN-201 may help make the protein needed to restore typical heart function
- TN-201 is given as a one-time intravenous (IV) infusion
What is the purpose of the MyPEAK-1 clinical trial?
MyPEAK-1 is a Phase 1b clinical trial. This means it is the first clinical trial evaluating TN-201 in humans. The goals are to understand:
- The safety of TN-201 in humans
- Any potential side effects
- The effects of TN-201 on the human body
- The best dose of TN-201
The MyPEAK-1 clinical trial will also look at the way TN-201 affects overall health and quality of life based on feedback from participants and their doctors. In this clinical trial, all participants will receive TN-201.
AAV gene therapy does carry some risk. Read about the known risks of AAV gene therapy here or ask your doctor for more information.
In collaboration with experts in HCM and gene therapy, the FDA and Tenaya carefully developed the plans for the MyPEAK-1 clinical trial. These plans are called the protocol. The protocol for the MyPEAK-1 clinical trial is similar to protocols used in other gene therapy clinical trials around the world.
Who can take part in the MyPEAK-1 clinical trial?
The MyPEAK-1 clinical trial will initially enroll a limited number of people in the United States and may expand to other countries in the future. Clinical trial site staff will evaluate people interested in taking part in MyPEAK-1 and determine if they are eligible.
To be eligible for this clinical trial, a person must:
- Be 18 to 65 years old
- Have HCM caused by a MYBPC3 gene mutation
- Have mild (Class II) or moderate (Class III) heart failure, according to New York Heart Association (NYHA) functional criteria
- Have a low level of antibodies to AAVs
These are not all of the eligibility criteria for this clinical trial.
If a person is not eligible for MyPEAK-1 clinical trial, there may be opportunities to take part in future Tenaya studies for people with genetic HCM that may have different eligibility criteria.
Participation in the MyPEAK-1 clinical trial is entirely voluntary. There is no cost to participate in this clinical trial. Participants can choose whether they want to take part in the study, and participants can change their mind at any time.
For more information about MyPEAK-1 clinical trial, talk to your doctor or contact clinical.trials@tenayathera.com or patient.advocacy@tenayathera.com.
- You can also visit ClinicalTrials.gov and enter identifier number NCT05836259
- Or share this MyPEAK-1 Clinical Trial Fact Sheet with your doctor.
What are the risks associated with AAV gene therapy?
Clinical trials of AAV gene therapies are still ongoing and not all of the risks are known. Known risks today include:
- The immune system may recognize the gene therapy as a harmful intruder. AAVs do not cause diseases in people. However, the immune system is designed to remove anything it does not recognize as part of a person’s body. The immune system may react by attacking the treatment before it has a chance to work.1,2
- Some studies of AAV and other forms of gene therapy show that immune system reactions may affect a person’s liver2
- Medicines that suppress or subdue the immune system are typically given before a person receives AAV gene therapy to help prevent or reduce this reaction3
- Currently, a person can receive AAV gene therapy only once; it cannot be re-dosed or administered in a larger dose at a later time.1,2
- After the first treatment with AAV gene therapy, a person’s immune system makes antibodies to the AAV capsid. If the body sees the same AAV again, it will attack it before it has a chance to work4
- AAV gene therapy may deliver a working gene to cell or tissue types that are not their intended target. This may cause illness or harm to other cells.1,4
- This is why the working gene is packaged with specific instructions which tell the gene what to do in target cells and reduces potential effects on other cells5
- Participation in an AAV gene therapy clinical trial may prevent an individual from participating in clinical trials for other AAV gene therapies in the future. Although many clinical trials of AAV gene therapies have been completed to date, ask your healthcare provider about the known risks of AAV gene therapy.
What to expect in the MyPEAK-1 clinical trial
(Up to 12 weeks)
Screening
to determine whether a person can participate
Informed Consent
to ensure a person understands:
- The purpose of the clinical trial
- All participants will receive TN-201, the investigational gene therapy
- Expectations for participation including required visits, procedures, and tests
- Potential risks and benefits
- Options for leaving the clinical trial, if desired
Pre-treatment Activites
- Visits (2 and 3) to gain information about a person’s health prior to receiving TN-201
- The clinical trial doctor will start a person on certain medicines before receiving TN-201. The doctor will review the reason these medicines are needed and any potential side effects
Infusion of TN-201
- The infusion of TN-201 typically takes 2 to 4 hours
- Hospitalization for 8 days for close monitoring, management, and treatment of any possible side effects
First-year follow-up visits
(about 19 visits post-hospitalization over the course of 1 year) to monitor safety and changes in heart function and HCM symptoms
Most visits will be in person at the trial site with a select number of visits completed by phone or at a local lab.
About 4 years
Long-term follow-up
(5 visits total, 1 per year) to monitor safety and changes in heart function and HCM symptoms over time
Participants can choose whether they want to take part in the clinical trial, and they can change their mind at any time.
Clinical trial locations
- Tenaya is working with leading experts in gene therapy and HCM to open clinical trial sites around the world.
- Visit the MyPEAK-1 clinical trial page on ClinicalTrials.gov (NCT05836259) for more information about the clinical trial and which centers are participating.
- Use the map below to search for participating centers.
United States Locations
California Locations
La Jolla, California, United States, 92093
Recruiting
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
Contact: Connor Davies
San Francisco, California, United States, 94117
Recruiting
University of California San Francisco
Contact: Emma Reagan
Georgia Locations
Atlanta, Georgia, United States, 30322
Recruiting
Emory University
Contact: Joey Freshwater
Massachusetts Locations
Boston, Massachusetts, United States, 02115
Recruiting
Brigham and Women's Hospital
Contact: Mary Sheehan
Contact: Alyx Vogle
Minnesota Locations
Rochester, Minnesota, United States, 55905
Recruiting
Mayo Clinic
Contact: Levi Disrud
Ohio Locations
Cincinnati, Ohio, United States, 45219
Recruiting
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
Contact: Kasey Riffey
Cleveland, Ohio, United States, 44195
Recruiting
Cleveland Clinic
Contact: Danielle Kellner
Oregon Locations
Portland, Oregon, United States, 97239
Recruiting
Oregon Health & Science University
Contact: Ahmad Masri, MD
Pennsylvania Locations
Philadelphia, Pennsylvania, United States, 19104
Recruiting
Hospital of the University of Pennsylvania
Contact: Kimberly Clinton
Texas Locations
Houston, Texas, United States, 77030
Recruiting
Houston Methodist Hospital
Contact: Jennifer Garrett
Scroll to see more locations
If you are interested in participating:
Talk to your doctor about whether you may be a candidate for this clinical trial
Visit ClinicalTrials.gov (NCT05836259) to learn more, including where the clinical trial is being conducted. Check back often as the list will be updated when new clinical trial sites are available
Contact clinical.trials@tenayathera.com or patient.advocacy@tenayathera.com to request more information
The use of TN-201 described here is investigational. Safety and efficacy have not been established. TN-201 has not been approved by the U.S. Food and Drug Administration or any other country’s health authority or regulatory agency.
Expand for References
1. Au HKE, et al. Gene therapy advances: a meta-analysis of AAV usage in clinical settings. Front Med. 2022;8:809118. https://doi.org/10.3389/fmed.2021.809118. 2. Delire B, et al. Immunotherapy and gene therapy: new challenges in the diagnosis and management of drug-induced liver injury. Front Pharmacol. 2022;12:786174. https://doi.org/10.3389/fphar.2021.786174. 3. Xiang Z, et al. The effect of rapamycin and ibrutinib on antibody responses to adeno-associated virus vector mediated gene transfer. Hum Gene Ther. 2022;33:614-624. https://doi.org/10.1089/hum.2021.258. 4. Gene Therapy. Mayo Foundation for Medical Education and Research; 2022. https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619. Accessed April 15, 2024. 5. Domenger C, Grimm D. Next-generation AAV vectors – do not judge a virus (only) by its cover. Human Molecular Genetics. 2019;28:R3-R14. https://doi.org/10.1093/hmg/ddz148.
