Exploratory Study of MYK-491 in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants

Title: 
Exploratory Study of MYK-491 in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants
Recruitment Status: 
Status Last Updated: 
October 1, 2020
Study Purpose: 

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.

Intervention/Treatment: 
Drug: danicamtiv
Phase: 
Study Description: 

N/A

Study Type: 
Official Title: 
Open-Label Exploratory Study of Oral MYK-491 in Stable Ambulatory Patients With Primary Dilated Cardiomyopathy Due to Either MYH7 or TTN Variants
Study Start Date: 
August 19, 2020
Study Completion Date: 
July 2021
Primary Objective(s): 

Frequency and severity of treatment-emergent adverse events and serious adverse events. [ Time Frame: Up to 22 days ]

Secondary Objective(s): 

N/A

Eligibility: 

Ages Eligible for Study:  18 Years to 80 Years   (Adult, Older Adult)

Sexes Eligible for Study:  All

Accepts Healthy Volunteers:  No

Inclusion Criteria: 

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

Exclusion Criteria: 
  • 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
Study Site(s)/Location(s): 
  • United States, Massachusetts
  • Brigham and Women's HospitalRecruiting
  • Boston, Massachusetts, United States, 02115
  • Contact: Mary Sheehan, RN    617-732-6237      
Sponsors & Collaborators: 

MyoKardia, Inc.

Principal Investigator(s): 

Myokardia Medical Information

650-741-0900

medinfo@myokardia.com

For more information, please contact the Study Coordinator: 

Contact:  

Email: 

Phone: 

ClinicalTrials.gov ID: 
NCT04572893