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The Sponsor is studying an investigational medication called danicamtiv to determine if it can help people with genetic and familial dilated cardiomyopathy (DCM). Investigational means that the safety and effectiveness of danicamtiv have not been established. Currently, there are no approved drugs that are designed specifically to treat genetic or familial DCM.
The purpose of this study is to evaluate how well danicamtiv works compared to a placebo (sugar pill that looks like danicamtiv pill but does not contain any danicamtiv) and see how safe it is for people with genetic and familial DCM. In DCM, the heart muscle weakens and enlarges, making it harder for the heart to pump blood; this can happen for different reasons. Some people have DCM because of a change in a gene (called genetic DCM). Others may have DCM that runs in their family, even if no specific gene change is found (called familial DCM).
The main goals of the study are:
Participants will:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| danicamtiv | Experimental |
| |
| placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| danicamtiv | Drug | Danicamtiv will be administrated twice daily for up to 26 weeks |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change from Baseline (Day 1) to Week 26 in left atrial function index in Cohort 1 | 26 weeks | |
| Part 2: Change from Baseline (Day 1) to Week 26 in peak VO2 in Cohort 1 | 26 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change from Baseline (Day 1) to Week 26 in peak VO2 | 26 weeks | |
| Part 1: Association between response in left ventricular global longitudinal strain and/or left atrial function index after 2 weeks of active treatment with placebo-adjusted Week 26 response in peak VO2 |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1 and 2: Incidence and severity of treatment emergent adverse events, adverse events of special interest, serious adverse events and disease related events | 26 weeks | |
| Incidence of abnormal findings in laboratory assessments, physical examinations, 12-lead ECGs, and vital signs |
Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kardigan Clinical Trial Information Team | Contact | 1-877-310-5135 | clinicaltrials@kardigan.bio |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama Birmingham | Recruiting | Birmingham | Alabama | 35233 | United States |
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| Placebo |
| Drug |
Placebo will be administrated twice daily for up to 26 weeks |
|
| 26 weeks |
| Part 1: Change from Baseline (Day 1) to Week 26 in left atrial function index in overall population | 26 weeks |
| Part 1 and 2: Change from Baseline (Day 1) to Week 26 in left ventricular ejection fraction | 26 weeks |
| Part 1 and 2: Change from Baseline (Day 1) to Week 26 in ventilatory efficiency | 26 weeks |
| Part 1 and 2: Change from Baseline (Day 1) to Week 26 in Kansas City Cardiomyopathy Questionnare (KCCQ-23) Clinical Summary Score | 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. | 26 weeks |
| Part 1: Proportion of participants from Baseline (Day 1) to Week 26 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. | 26 weeks |
| Part 2: Change from Baseline (Day 1) to Week 26 in left atrial function index | 26 weeks |
| Part 2: Change from Baseline (Day 1) to Week 26 in peak VO2 in the responder subgroup | 26 weeks |
| Part 2: Change from Baseline (Day 1) to Week 26 in peak VO2 in overall population | 26 weeks |
| Part 1 and 2: Change from Baseline (Day 1) to Week 26 in left ventricular global longitudinal strain | 26 weeks |
| Part 1 and 2: Percent change from Baseline (Day 1) to Week 26 in NT-proBNP concentrations | 26 weeks |
| Part 1 and 2: Characterization of peak (Cmax) of danicamtiv | 20 weeks |
| Part 1 and 2: Characterization of trough (Cmin) plasma concentrations of danicamtiv | 20 weeks |
| 26 weeks |
| Cedars Sinai | Recruiting | Los Angeles | California | 90048 | United States |
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| University of California, San Diego (UCSD) - Medical Center | Recruiting | San Diego | California | 92037 | United States |
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| UCSF | Recruiting | San Francisco | California | 94158 | United States |
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| MedStar Washington Hospital Center | Recruiting | Washington D.C. | District of Columbia | 20010 | United States |
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| Mayo Clinic - Jacksonville | Recruiting | Jacksonville | Florida | 32224 | United States |
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| Northwestern University | Recruiting | Chicago | Illinois | 60611 | United States |
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| Louisiana State University (LSU) Health Sciences Center | Recruiting | New Orleans | Louisiana | 70112 | United States |
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| Brigham and Women's Hospital | Recruiting | Boston | Massachusetts | 02115 | United States |
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| Henry Ford Health System | Recruiting | Detroit | Michigan | 48202 | United States |
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| University of Minnesota | Recruiting | Minneapolis | Minnesota | 55455 | United States |
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| Mayo Clinic Rochester | Recruiting | Rochester | Minnesota | 55905 | United States |
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| Washington University in St. Louis | Recruiting | St Louis | Missouri | 63110 | United States |
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| University of Nebraska Medical Center | Recruiting | Omaha | Nebraska | 68106 | United States |
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| NYU Langone Health | Recruiting | New York | New York | 10016 | United States |
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| Columbia University Medical Center/ New York Presbyterian Hospital | Not yet recruiting | New York | New York | 10027 | United States |
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| Duke University Medical Center | Recruiting | Durham | North Carolina | 27708 | United States |
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| The Lindner Center for Research and Education at The Christ Hospital | Recruiting | Cincinnati | Ohio | 45219 | United States |
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| The Ohio State University Wexner Medical Center | Recruiting | Columbus | Ohio | 43210 | United States |
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| Oregon Health & Science University (OHSU) | Recruiting | Portland | Oregon | 97239 | United States |
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| University of Pennsylvania | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| Vanderbilt University Medical Center | Recruiting | Nashville | Tennessee | 37232 | United States |
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| Baylor College of Medicine | Recruiting | Houston | Texas | 77030 | United States |
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| Universite Libre de Bruxelles (ULB) - Hopital Erasme | Recruiting | Brussels | 1070 | Belgium |
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| Universitair Ziekenhuis Antwerpen (UZA) | Recruiting | Edegem | 2650 | Belgium |
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| Aarhus University Hospital | Recruiting | Aarhus | 8200 | Denmark |
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| Rigshospitalet | Recruiting | Copenhagen | 1050 | Denmark |
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| Odense University Hospital - Odense | Recruiting | Odense | 5000 | Denmark |
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| AP-HP Hopital Pitie-Salpetriere | Recruiting | Paris | 75013 | France |
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| AP-HP Hopital Europeen Georges Pompidou | Recruiting | Paris | 75015 | France |
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| Semmelweis Egyetem - Varosmajori Sziv es Ergyogyaszati Klinika | Recruiting | Budapest | 1122 | Hungary |
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| Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont | Recruiting | Szeged | 6724 | Hungary |
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| Azienda Ospedaliero-Universitaria Careggi | Recruiting | Florence | 50134 | Italy |
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| Istituto Clinico Humanitas | Recruiting | Rozzano | 20089 | Italy |
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| Azienda Sanitaria Universitaria Giuliano Isontina (ASU GI) - Ospedale di Cattinara | Recruiting | Trieste | 34100 | Italy |
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| Amsterdam UMC - Locatie AMC | Recruiting | Amsterdam | 1105-AZ | Netherlands |
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| Erasmus MC | Recruiting | Rotterdam | 3015 GD | Netherlands |
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| Krakowski Szpital Specjalistyczny im. sw. Jana Pawla II | Recruiting | Krakow | 31-202 | Poland |
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| Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi | Recruiting | Lodz | 92-213 | Poland |
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| Hospital Universitario Vall d'Hebron | Recruiting | Horta-Guinardó | Barcelona | 08035 | Spain |
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| Hospital Universitario Puerta de Hierro | Recruiting | Majadahonda | Madrid | 20222 | Spain |
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| Hospital Universitario de A Coruña | Recruiting | A Coruña | 15006 | Spain |
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| Hospital Universitari de Bellvitge | Recruiting | Barcelona | 08907 | Spain |
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| Hospital Universitario Virgen de la Victoria | Recruiting | Málaga | 29001 | Spain |
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| Hospital Clinico Universitario Virgen de la Arrixaca | Recruiting | Murcia | 30120 El Palmar, | Spain |
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| Hospital Universitario Son Llatzer | Recruiting | Palma de Mallorca | 7198 | Spain |
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| Hospital Universitario Virgen del Rocio | Recruiting | Seville | 41013 | Spain |
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| Sodersjukhuset AB | Recruiting | Stockholm | 11883 | Sweden |
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| University Hospitals Bristol NHS Foundation Trust | Recruiting | Bristol | BS1 3NU | United Kingdom |
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| NHS Greater Glasgow and Clyde | Recruiting | Glasgow | G12 0YN | United Kingdom |
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| Glenfield Hospital | Recruiting | Leicester | LE3 9QP | United Kingdom |
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| St. Bartholomew's Hospital | Recruiting | London | EC1A | United Kingdom |
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| St George's Hospital | Recruiting | London | SW17 0QT | United Kingdom |
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| Royal Brompton Hospital | Recruiting | London | SW3 6NP | United Kingdom |
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| Hammersmith Hospital, Imperial College of London | Recruiting | London | W12 0HS | United Kingdom |
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| Cardiovascular Clinical Research Facility (CCRF) | Recruiting | Oxford | OX3 9DU | United Kingdom |
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