A Study to Evaluate MYK-491 to Treat Patients with Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants


About this study

The purpose of this study is to establish safety and preliminary effectiveness of treatment with MYK-491 in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants .

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- For MYH7 and TTN cohorts, must have diagnosis of primary DCM (dilated cardiomyopathy),
clinically stable and due to probably disease-causing variant of MYH7 or TTN

- Has adequate acoustic windows for echocardiography

- Maximum of 3 family members with same variant can be enrolled

- For the cohort of primary DCM due to causalities other than MYH7 and TTN, participant
must have diagnosis of primary DCM with a cause not related to MYH7 or TTN variants

Exclusion Criteria:

- Significant structural cardiac abnormalities including valvar dysfunction on Screening
transthoracic echo(s)

- Routinely scheduled outpatient intravenous (IV) infusions for heart failure (e.g.,
inotropes, afterload reduction, or diuretics)

- Presence of protocol specified laboratory abnormalities at Screening

- Recent acute coronary syndrome or angina pectoris (<90 days)

- Recent hospitalization for heart failure (<90 days)

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 4/12/23. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Naveen Pereira, M.D.

Open for enrollment

Contact information:

Susan Milbrandt C.C.R.C.

(507) 538-6105


More information


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