Back to resultsStudy Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-461
Primary Purpose
Hypertrophic Cardiomyopathy
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
MYK-461
Sponsored by
About this trial
This is an interventional treatment trial for Hypertrophic Cardiomyopathy
Eligibility Criteria
Inclusion Criteria:
- Diagnosed with HCM
- Normal left ventricular ejection fraction (LVEF)
- NYHA class I, II or III
Exclusion Criteria:
- Inherited metabolic disorders, myocardial infiltration or cardiofaciocutaneous syndrome (e.g., Noonan's, Fabry's).
- History of clinically important atrial or ventricular arrhythmias
- History of positive human immunodeficiency virus (HIV) test and/or seropositive for hepatitis C virus (HCV) or hepatitis B virus (HBV)..
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
MYK-461
Arm Description
Outcomes
Primary Outcome Measures
Safety: Incidence of adverse events
Secondary Outcome Measures
Determination of pharmacokinetics parameters as measured by Cmax
maximum concentration (Cmax)
Determination of pharmacokinetics parameters as measured by Tmax
time of the maximum measured concentration (Tmax)
Determination of pharmacokinetics parameters as measured by AUC
area under the concentration-time curve (AUC)
Determination of pharmacokinetics parameters as measured by t1/2
half-life (t1/2)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02329184
Brief Title
Study Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-461
Official Title
Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-461 in Patient Volunteers With Hypertrophic Cardiomyopathy
Study Type
Interventional
2. Study Status
Record Verification Date
September 2016
Overall Recruitment Status
Completed
Study Start Date
December 2014 (undefined)
Primary Completion Date
March 2016 (Actual)
Study Completion Date
April 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MyoKardia, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to establish initial safety, tolerability, pharmacokinetics and pharmacodynamics of MYK-461 in human subjects. This is a sequential group, single ascending (oral) dose study in NYHA Class I, II, or III patient volunteers aged 18-65 years.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypertrophic Cardiomyopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
MYK-461
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
MYK-461
Primary Outcome Measure Information:
Title
Safety: Incidence of adverse events
Time Frame
28 Days
Secondary Outcome Measure Information:
Title
Determination of pharmacokinetics parameters as measured by Cmax
Description
maximum concentration (Cmax)
Time Frame
28 Days
Title
Determination of pharmacokinetics parameters as measured by Tmax
Description
time of the maximum measured concentration (Tmax)
Time Frame
28 Days
Title
Determination of pharmacokinetics parameters as measured by AUC
Description
area under the concentration-time curve (AUC)
Time Frame
28 Days
Title
Determination of pharmacokinetics parameters as measured by t1/2
Description
half-life (t1/2)
Time Frame
28 Days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosed with HCM
Normal left ventricular ejection fraction (LVEF)
NYHA class I, II or III
Exclusion Criteria:
Inherited metabolic disorders, myocardial infiltration or cardiofaciocutaneous syndrome (e.g., Noonan's, Fabry's).
History of clinically important atrial or ventricular arrhythmias
History of positive human immunodeficiency virus (HIV) test and/or seropositive for hepatitis C virus (HCV) or hepatitis B virus (HBV)..
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jonathan C Fox, MD, PhD
Organizational Affiliation
MyoKardia, Inc.
Official's Role
Study Director
Facility Information:
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
City
Cypress
State/Province
California
ZIP/Postal Code
90630
Country
United States
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49007
Country
United States
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Study Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-461
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