Symptomatic Genetic Dilated Cardiomyopathy (DCM)
Conditions
Brief summary
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.
Detailed description
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
Interventions
DRUGDanicamtiv
Danicamtiv will be administered twice daily for up to 5 years
Sponsors
Kardigan, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Global, multicenter study of danicamtiv in participants with symptomatic genetic or familial DCM who previously completed Part 1 or Part 2 of DAN-301 study. Once eligibility is confirmed, participants will receive danicamtiv twice daily dosing.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Completed 24 weeks of blinded study treatment (corresponding to the Week 26 Visit in DAN-301) and planned assessments in Part 1 or Part 2 of the DAN-301 study. 2. If sexually active, must use a highly effective birth control method from the Screening Visit through 3 months after the last dose. 3. Is able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to local and institutional guidelines before the first study-specific procedure.
Exclusion criteria
1. Acute coronary syndrome or hemodynamically significant epicardial coronary disease, or coronary revascularization (percutaneous coronary intervention \[PCI\] or coronary artery bypass graft \[CABG\]) performed within 90 days of DAN-302 Screening Visit. 2. Recent (\<90 days) hospitalization for heart failure (HF) or use of IV diuretic. 3. Other recent (\<90 days) cardiovascular events (e.g., cerebrovascular accident). 4. Hospitalization for sepsis or other systemic infections within 30 days of Screening. A systemic infection includes suspected or confirmed infection associated with fever, hemodynamic instability, bacteremia, or organ dysfunction typically requires urgent evaluation and/or acute care. 5. Any malignancy requiring treatment after enrollment in DAN-301 that could limit patient's survival.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency and severity of treatment-emergent adverse events (TEAEs) | 61 months |
| Frequency and severity of adverse events of special interest (AESIs) | 61 months |
| Frequency and severity of serious adverse events (SAEs) | 61 months |
| Frequency and severity of disease-related events (DREs) | 61 months |
| Frequency of abnormal findings from physical examination | 61 months |
| Frequency of abnormal findings from vital signs | 61 months |
| Frequency of abnormal findings from 12-lead electrocardiograms (ECGs) | 61 months |
| Frequency of abnormal findings from clinical laboratory assessments | 61 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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) | 61 months | 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) |
| 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) | 61 months | 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. |
| 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 | 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 | 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 | 61 months | 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. |
| 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 | 61 months | — |
Countries
Belgium, Denmark, France, Hungary, Italy, Netherlands, Poland, Spain, Sweden, United Kingdom, United States
Contacts
CONTACTKardigan Clinical Trial Information Team
Outcome results
None listed