Danicamtiv is an investigational medication, which means it is still being studied and has not been approved. The purpose of this study is to learn how well danicamtiv works, how safe it is, how well people can take it when used in patients with genetic or familial dilated cardiomyopathy (DCM). There are currently no approved medicines made specifically to treat genetic or familial DCM.
The Sponsor is studying an investigational medication called danicamtiv to understand its long-term benefits and risks in patients with symptomatic genetic or familial DCM. This study will examine the durability of treatment effects with continued exposure and evaluate appropriateness of chronic dosing over time. Participants will: * Take the investigational medicine, danicamtiv, twice daily throughout the study, which is up to 5 years * Visit the clinic about 16 times for clinical evaluations and diagnostic tests
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
332
Danicamtiv will be administered twice daily for up to 5 years
Frequency and severity of treatment-emergent adverse events (TEAEs)
Time frame: 61 months
Frequency and severity of adverse events of special interest (AESIs)
Time frame: 61 months
Frequency and severity of serious adverse events (SAEs)
Time frame: 61 months
Frequency and severity of disease-related events (DREs)
Time frame: 61 months
Frequency of abnormal findings from physical examination
Time frame: 61 months
Frequency of abnormal findings from vital signs
Time frame: 61 months
Frequency of abnormal findings from 12-lead electrocardiograms (ECGs)
Time frame: 61 months
Frequency of abnormal findings from clinical laboratory assessments
Time frame: 61 months
Change from Baseline (Day 1) to Week 26, Months 18, 30, 42, 54, 60, and 61 in cardiac structure, function, and size using Transthoracic Echocardiogram (TTE)
Including but not limited to left atrial function index (LAFI), left atrial volume index (LAVI), maximum left atrial volume index (LAmaxVi), minimum left atrial volume index (LAminVi), left atrial emptying fraction (LAEF), left ventricular ejection fraction (LVEF), left ventricular end-diastolic diameter (LVEDD), left ventricular end diastolic volume (LVEDV), left ventricular end systolic volume (LVESV), early diastolic mitral inflow velocity/early diastolic mitral annular tissue velocity (E/e'), left ventricular global longitudinal strain (LVGLS), left ventricular global circumferential strain (LVGCS), and left ventricular mass index (LVMI)
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University of Alabama Birmingham
Birmingham, Alabama, United States
RECRUITINGCedars Sinai
Los Angeles, California, United States
NOT_YET_RECRUITINGUniversity of California, Los Angeles (UCLA) - David Geffen School of Medicine
Los Angeles, California, United States
NOT_YET_RECRUITINGUniversity of California, San Diego (UCSD) - Medical Center
San Diego, California, United States
NOT_YET_RECRUITINGUniversity of California, San Francisco (UCSF)
San Francisco, California, United States
NOT_YET_RECRUITINGStanford University Medical Center
Stanford, California, United States
NOT_YET_RECRUITINGMedStar Washington Hospital Center
Washington D.C., District of Columbia, United States
NOT_YET_RECRUITINGMayo Clinic - Jacksonville
Jacksonville, Florida, United States
NOT_YET_RECRUITINGNorthwestern University
Chicago, Illinois, United States
NOT_YET_RECRUITINGLouisiana State University (LSU) Health Sciences Center
New Orleans, Louisiana, United States
NOT_YET_RECRUITING...and 68 more locations
Time frame: 61 months
Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS)
All scores are represented on a 0-to-100-point scale, where lower scores represent more severe symptoms and/or limitations and scores of 100 indicate no symptoms, no limitations, and excellent quality of life.
Time frame: 61 months
Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in EuroQol 5-Dimension 5-Level Questionnaire (EQ-5D-5L) Utility Score
Time frame: 61 months
Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in EuroQol Visual Analogue Scale (EQ-VAS) Score
Time frame: 61 months
Proportion of participants from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 who achieve at least a one-class improvement in New York Heart Association (NYHA) class
NYHA is a system used to categorize the severity of heart failure symptoms based on a patient's functional capacity, where 1 indicates no symptoms or limitations and 4 indicates symptoms present at rest.
Time frame: 61 months
Change from Baseline (Day 1) to Week 26, Months 12, 18, 24, 30, 36, 42, 48, 54, 60, and 61 in N-terminal pro-B-type natriuretic peptide (NT-proBNP) concentrations
Time frame: 61 months