Primary Familial Dilated Cardiomyopathy Clinical Trial
Official title:
An Open-Label, Exploratory Study of the Safety and Preliminary Efficacy of Danicamtiv in Stable Ambulatory Participants With Primary Dilated Cardiomyopathy Due to Either MYH7 or TTN Variants or Other Causalities
| Verified date | March 2024 |
| Source | Bristol-Myers Squibb |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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 or other causalities.
| Status | Terminated |
| Enrollment | 41 |
| Est. completion date | February 22, 2024 |
| Est. primary completion date | February 22, 2024 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 80 Years |
| Eligibility | Inclusion Criteria: - For MYH7 and TTN cohorts, must have diagnosis of primary DCM (dilated cardiomyopathy), clinically stable and due to probably disease-causing variant of MYH7 or TTN - Has adequate acoustic windows for echocardiography - Maximum of 3 family members with same variant can be enrolled - For the cohort of primary DCM due to causalities other than MYH7 and TTN, participant must have diagnosis of primary DCM with a cause not related to MYH7 or TTN variants Exclusion Criteria: - Significant structural cardiac abnormalities including valvar dysfunction on Screening transthoracic echo(s) - 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) |
| Country | Name | City | State |
|---|---|---|---|
| Germany | Local Institution - 0014 | Heidelberg | |
| Germany | Local Institution - 0015 | Wuerzburg | |
| Spain | Local Institution - 0012 | A Coruña | |
| Spain | Local Institution - 0011 | El Palmar | |
| Spain | Local Institution - 0013 | Majadahonda | |
| United Kingdom | Local Institution - 0016 | London | |
| United Kingdom | Local Institution - 0017 | Middlesex | |
| United States | Local Institution - 0018 | Austin | Texas |
| United States | Local Institution - 0003 | Boston | Massachusetts |
| United States | Local Institution - 0004 | Charleston | South Carolina |
| United States | Local Institution - 0002 | Chicago | Illinois |
| United States | Local Institution - 0006 | Cleveland | Ohio |
| United States | Local Institution - 0019 | Columbus | Ohio |
| United States | Local Institution - 0008 | Germantown | Tennessee |
| United States | Local Institution - 0007 | La Jolla | California |
| United States | Local Institution - 0001 | Philadelphia | Pennsylvania |
| United States | Local Institution - 0005 | Rochester | Minnesota |
| United States | Local Institution - 0009 | Tampa | Florida |
| United States | Local Institution - 0010 | Washington | District of Columbia |
| Lead Sponsor | Collaborator |
|---|---|
| Bristol-Myers Squibb |
United States, Germany, Spain, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Frequency and severity of treatment-emergent adverse events and serious adverse events. | Up to 22 days, optional extension of up to 96 weeks | ||
| Primary | Number of participants with vital sign abnormalities | Up to 22 days, optional extension of up to 96 weeks | ||
| Primary | Number of participants with adverse event abnormalities | Up to 22 days, optional extension of up to 96 weeks | ||
| Primary | Number of participants with physical examination abnormalities | Up to 22 days, optional extension of up to 96 weeks | ||
| Primary | Number of participants with ECG abnormalities | Up to 22 days, optional extension of up to 96 weeks | ||
| Primary | Number of participants with safety lab abnormalities | Up to 22 days, optional extension of up to 96 weeks | ||
| Secondary | Change in pharmacodynamic (PD) parameters assessed by transthoracic echocardiography (TTE) | Parameters include: Left ventricular systolic ejection time Left ventricular stroke volume Left ventricular ejection fraction Left ventricular global longitudinal strain Left ventricular global circumferential strain Tissue Doppler imaging (TDI) of mitral valve annulus peak systolic velocity (s') Left ventricular end systolic diameter Left ventricular end diastolic diameter Left ventricular end diastolic volume, indexed for body surface area (BSA) Left ventricular end systolic volume, indexed for BSA Left atrial maximum volume, indexed for BSA Left atrial minimum volume, indexed for BSA Left atrial emptying fraction Left atrial function index TDI of mitral valve annulus peak velocity in diastole (e', lateral, septal) Ratio of peak inflow velocities in early and late diastole Ratio of early mitral peak inflow velocity to early mitral peak annulus velocity (TDI) (E/e') lateral, septal, and average |
Up to 96 weeks |