Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants
Clinicaltrials.gov identifier recruitment status First Posted Last update posted
NCT04572893 Recruiting October 1, 2020 November 19, 2021

study description
Brief Summary

The purpose of this Phase 2a study is to establish safety and preliminary efficacy of treatment with danicamtiv in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants .

Condition or Disease: Primary Familial Dilated Cardiomyopathy
Intervention/treatment: Drug: danicamtiv
Phase: Phase 2
Detailed Description

N/A


study design
Study Type: Interventional
Estimated Enrollment : 24 participants
Intervention Model : Sequential Assignment
Masking: None (Open Label) ()
Primary Purpose: Treatment
Official Title: An Open-Label, Exploratory Study of the Safety and Preliminary Efficacy of Danicamtiv in Stable Ambulatory Patients With Primary Dilated Cardiomyopathy Due to Either MYH7 or TTN Variants
Actual Study Start Date: August 2020
Estimated Primary Completion Date: March 2022
Estimated Study Completion Date: March 2024

Arms and interventions
Arm Intervention/treatment
Experimental: MYK-491
Primary DCM due to MYH7 or TTN Variant
Drug: danicamtiv
Myosin activator
outcome measures
Primary Outcome Measures: 1. Frequency and severity of treatment-emergent adverse events and serious adverse events. [ Time Frame: Up to 22 days, optional extension of up to 96 weeks ]

Eligibility Criteria
Ages Eligible for Study: 18 to 80 Years (Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria

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:

Significant structural cardiac abnormalities including valvar dysfunction 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, 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)


Contacts and Locations
Contacts

Contact: BMS Medical Information (Use email contact) clinical.trials@bms.com

Locations
United States, California University of California, San Diego Medical Center La Jolla
United States, District of Columbia MedStar Georgetown University Hospital Washington
United States, Florida University of South Florida Tampa
United States, Illinois Northwestern Medical Faculty Foundation Chicago
United States, Massachusetts Brigham and Women's Hospital Boston
United States, Minnesota Mayo Clinic - Rochester Rochester
United States, Ohio Cleveland Clinic Cleveland
United States, Ohio Ohio State University Medical Center Columbus
United States, Pennsylvania University of Pennsylvania - Heart and Vascular Center Philadelphia
United States, South Carolina Medical University of South Carolina Charleston
United States, Tennessee The Stern Cardiovascular Center Germantown
United States, Texas Austin Heart Austin
Germany Universitaetsklinikum Heidelberg Heidelberg
Germany Universitaetsklinikum Würzburg Würzburg
Spain, Madrid Hospital Universitario Puerta de Hierro Majadahonda Majadahonda
Spain, Murcia Hospital Clinico Universitario Virgen de la Arrixaca El Palmar
Spain Complejo Hospitalario Universitario A Coruña A Coruña
United Kingdom St. Bartholomew's Hospital London
United Kingdom Royal Brompton Hospital London
Sponsors and Collaborators
MyoKardia, Inc.
Investigator
More Information
Responsible Party : MyoKardia, Inc.
ClinicalTrials.gov Identifier : NCT04572893     
Other Study ID Numbers : MYK-491-006, 2019-003626-24
First Posted : October 1, 2020
Last Update Posted : November 19, 2021
Last Verified : November 2021
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by MyoKardia, Inc.: Primary dilated cardiomyopathy (DCM)
Familial dilated cardiomyopathy (DCM)
Myosin Heavy Chain 7 (MYH7)
Titin (TTN)
MYK-491
danicamtiv
Additional relevant MeSH terms :
Cardiomyopathies
Cardiomyopathy, Dilated
Heart Diseases
Cardiovascular Diseases
Cardiomegaly
Laminopathies
Genetic Diseases, Inborn