Gene Therapy Trial for ACM

Lexeo Gene Therapy Trial for Arrythmogenic Cardiomyopathy

Research alert!

Lexeo recently began enrolling participants for a gene therapy clinical trial for PKP2 arrhythmogenic cardiomyopathy (called ARVC or ACM).

The trial, called HEROIC-PKP2, will be conducted at multiple sites in the US and is enrolling adults ages 18 to 65.

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

In our bodies, there is a gene called plakophilin-2 (PKP2) that gives instructions to make an essential protein for the structure and function of the heart muscle. If you have ARVC/ACM with a PKP2 genetic variant it leads to a decreased amount of plakophilin-2 protein. This causes cells in the body, including heart muscle cells, to work abnormally. In ARVC/ACM, the heart muscle can weaken, which can lead to heart failure and abnormal heart rhythms.

This investigational gene therapy, called LX2020, aims to deliver a working PKP2 gene to heart cells to restore PKP2 protein levels, and to potentially improve the outcome for patients with PKP2 ARVC/ACM. The main goal of this trial is to learn about the safety and tolerability of 2 different doses of this investigational gene therapy. All trial participants will receive a one-time intravenous injection (into the bloodstream via needle) of LX2020.

Learn more about gene therapy trials – including FAQs, how gene therapy are different from other clinical trials, and questions you may have as you consider whether participation is right for you here.

What are the basic eligibility criteria?

  • Participants must be 18 to 65 years of age.
  • Adults with clinical diagnosis or arrythmogenic cardiomyopathy (ARVC/ACM)
  • Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2. That means they must have been genetically tested. (If you have been diagnosed with ARVC/ACM 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.

FAQs

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

Taking part in this trial is voluntary. This means that you are the one that can choose if you want to take part in the trial. Once you enroll in the trial, you can leave the trial at any time for any reason.

Will I be able to continue taking my current treatment?

Participants should discuss continuation of current treatments or medications with the study doctor.

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

Participants who are eligible and participate in this study will receive the investigational gene therapy and can expect regular office visits during the study period. There will also be several visits over the next four years as part of the long-term follow-up period. Details can be discussed with the study doctor.

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

Yes! The investigational gene therapy and all study-related assessments will be provided at no cost, and you will be reimbursed for reasonable study-related expenses, which may include fees for parking, meals, and other reasonable accommodations. Travel and accommodation will be arranged (and the cost covered) for participants in the study and one caregiver.

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