RIDGE-1 Gene Therapy Trial for PKP2-ARVC

ARVC: Tenaya RIDGE TM-1 Gene Therapy Trial for PKP2-ARVC

 

Currently Enrolling

Phase 1b

Sponsor: Tenaya Therapeutics

Tenaya is currently enrolling participants in the RIDGE™-1 clinical trial to study TN-401 gene therapy trial for PKP2-associated arrhythmogenic cardiomyopathy (ARVC, also known as ACM/ARVD).

RIDGE™ -1 is the first trial to study TN-401 (a gene therapy) in humans. Almost half of all ARVC cases are caused by genetic mutations. Mutations (also known as variations or changes) in the plakophilin-2 (PKP2) gene can prevent heart muscle cells from making enough of necessary proteins to support the structure and communication of heart muscle cells. TN-401 is an investigational gene therapy designed to deliver a working PKP2 gene to heart muscle cells and help make the protein needed to restore typical heart function.

The RIDGE-1 clinical trial will assess the safety of TN-401 and look at how it affects overall health and quality of life based on feedback from participants and their doctors. The goals are to understand:

  • How safe TN-401 is for humans
  • Any potential side effects
  • The best dose of TN-401
  • The effects of TN-401 on the human body

Basic eligibility criteria

  • Participants must be 18 to 65 years of age.
  • Clinical diagnosis of ARVC.
  • Patient must have an ICD implanted at least 6 months prior to screening.
  • Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2. If you have been diagnosed with ARVC but haven’t had genetic testing, SADS can help you with next steps. For resources on genetic testing click here to learn more, or email [email protected]for help.)

Full eligibility criteria will be evaluated by the clinical trial doctor to determine if an individual is the right candidate for this trial. If a person is not eligible for RIDGE-1 clinical trial, there may be opportunities to take part in future Tenaya studies for people with genetic ARVC that may have different eligibility criteria.

FAQs

What is the gene therapy being investigated, and how does it work?

TN-401 is an investigational gene therapy designed to replace the mutated gene with a working PKP2 gene to address the underlying causes of ARVC. Mutations in the PKP2 gene can prevent the heart muscle cells from making enough of the PKP2 protein, which works with other proteins to support the structure and communication of heart muscle cells.

  • TN-401 uses an adeno-associated virus (AAV) as a capsid (also called a vector) as a vehicle to deliver a working gene to the muscle cells of the heart. 
  • Once in these cells, the working gene delivered by TN-401 may help make the protein needed to restore typical heart function
  • TN-401 is given as a one-time intravenous (IV) infusion                                                                                                                   

Learn more about gene therapy trials – including FAQs, how gene therapy is different from other clinical trials, risks associated with AAV gene therapy and questions you can ask as you consider whether participation is right for you here

TN-401 has not been approved by the FDA or any other regulatory authority.

What if I begin the trial, and decide I would like to stop?

Participation in the RIDGE-1 clinical trial is entirely voluntary. Participants can choose whether they want to take part in the study, and participants can change their mind at any time.

Where are the clinical trial sites and how often would I need to travel there?

Participation in this study will involve attending study visits over an approximate 1-year period, followed by a long-term follow-up period of 4 years.

Current clinical trial site locations enrolling participants are listed below.  Additional clinical trial sites in the US will open soon. If you are interested in hearing from us when other locations begin enrolling, please register below.

  • UCSF, San Francisco, California, United States
  • U of Colorado, Aurora, Colorado, United States
  • Brigham and Women’s Hospital Boston, Massachusetts, United States
  • Mayo Clinic, Rochester, Minnesota, United States

Learn more about these sites here.

Will the costs of participating in the trial be covered by Tenaya?

All study-related testing and medications are free. A complementary travel service is provide by Tenaya to coordinate and book travel, provide a prepaid card for meals, and reimburse for parking and transportation fees for the patient and the patient’s caregiver.  Tenaya is able to provide support with hotel options as well. Patients should plan to stay close to the trial site at the beginning of the trial for several weeks.

The content for this webpage was developed by the SADS Foundation.

Please fill out this form if you are interested in learning more about this trial – including your eligibility – by connecting to a study coordinator. Once you have filled out the form, a staff member at SADS will connect you with the study coordinator at the site you’ve indicated below.

The information contained herein is being made available as a public service by the SADS Foundation (SADS). No posted information or material provided is intended to constitute medical or professional advice. SADS makes no representations or warranties, either express or implied, as to the accuracy of any posted information and assumes no responsibility for any errors or omissions contained therein. SADS makes no representations or warranties, either express or implied, that any products or services adhere to or satisfy any standards or requirements that may be applicable to such product or service. Furthermore, no warranty, express or implied, is created by providing information and does not in any way constitute an endorsement by SADS. No one shall be entitled to claim detrimental reliance on any views or information, or to claim any duty on the part of SADS to update posted information. In no event shall SADS be held liable to you or anyone else for any decision made or action taken in reliance on such information or view.