Current Clinical Trials & Research Studies

Overview

Research studies and clinical trials are carefully designed to evaluate the safety and effectiveness of potential treatments. Gene therapy clinical trials for cardiomyopathy provide patients the possibility of access to an investigational treatment and the ability to contribute to the advancement of scientific research that may also help other individuals with the disease. 

To learn more about Gene Therapy Clinical Trials and how they work, click here.

Current Genetic Cardiomyopathy Clinical Trials

PKP2 Clinical Trial (Click to learn more)

Lexeo’s HEROIC-PKP2 Clinical Trial – for eligible patients with confirmed ACM (also called ARVC) caused by the PKP2 genetic mutation.

About HEROIC-PKP2: ● The trial is open to individuals aged 18 to 65 with genetically confirmed ACM due to a PKP2 pathogenic variant.

Full eligibility criteria will be evaluated by the clinical trial doctor to determine if an individual is the right candidate for this trial. ● The main goal of this trial is to learn about the safety and tolerability of different doses of the investigational gene therapy LX2020. All trial participants will receive a one-time intravenous injection of LX2020. ● The investigational gene therapy and all trial-related assessments will be provided at no cost to study participants.

Reasonable trial-related expenses such as fees for parking and meals will also be covered by the study sponsor, Lexeo. Travel and accommodation will be arranged for participants in the trial and one caregiver.

To learn more about this clinical trial, please visit: https://patients.lexeotx.com/clinical-trials/lx2020/

MYBPC3 Clinical Trial (Click to learn more)

Tenaya’s MyPEAK-1 Clinical Trial – for people with MYBPC3-associated HCM.

Who can take part in the MyPEAK-1 clinical trial? To be eligible for this clinical trial, a person must: ● Be 18 to 65 years old ● Have nonobstructive 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 working implantable cardiac defibrillator (ICD)

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 can change their minds at any time.

To learn more about this clinical trial, please visit HCMStudies.com.

Genetic Cardiomyopathy Clinical Trials Finder

For a more complete listing of genetic cardiomyopathy research studies and clinical trials, please visit www.clinicaltrials.gov.

There, you will find a search tool that allows you to search current clinical trials available for your condition. (See sample search tool instructions and image below.)

Please follow these instructions to use the search tool:

  1. In the box labeled “Condition/disease”, simply type in “cardiomyopathy.”
    • Alternatively, you can type in “DCM”, “HCM”, or “ARVC”.
    • You can also type in your specific gene mutation to locate clinical trials associated with your gene.
    • Or, if you know the trial’s “NCT” number, you can enter it and you will be taken directly to that trial page.
  2. In the box labeled “Other terms” you can also type in a company name if you know of a company doing a clinical trial in your disease area. Or, you can type in the name of a particular study drug, or any other term that will help define your search.
  3. Location: Initially, we recommend leaving the location field blank.

You may be surprised at the number of clinical trials currently underway for various types of genetic cardiomyopathy. This tool allows you to be more selective in how you search. Please use the filters to narrow search results to those trials that may best suit your particular situation.

Other Resources


Please note:

The DCM Foundation and the Genetic Cardiomyopathy Awareness Consortium do not endorse or encourage you to participate in any specific research study or therapy clinical trial. Given our disease has no known cure(s) we aim to provide this information to our patients to review and decide independently if they would like to participate.

If you are considering participating in a research study or clinical trial please consult your medical team.

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