The purpose of this Phase 2a study is to establish safety and tolerability of treatment with MYK-491 in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants .
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Frequency and severity of treatment-emergent adverse events and serious adverse events. [ Time Frame: Up to 22 days ]
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Ages Eligible for Study: 18 Years to 80 Years (Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Has stable primary dilated cardiomyopathy due to either MYH7 or TTN variant
Has adequate acoustic windows for echocardiography
Maximum of 3 family members with same variant can be enrolled
Any significant structural cardiac abnormalities on Screening transthoracic echo(s)
A pathogenic variant implicated in DCM of another gene other than MYH7 or TTN
Routinely scheduled outpatient intravenous (IV) infusions for heart failure (e.g., inotropes, vasodilators [e.g., nesiritide], diuretics), or routinely scheduled ultrafiltration
Presence of protocol specified laboratory abnormalities at Screening
United States, Massachusetts
Brigham and Women's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Mary Sheehan, RN 617-732-6237
MyoKardia, Inc.
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