Study Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-461

Hypertrophic Cardiomyopathy Clinical Trial

Official title:

Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-461 in Patient Volunteers With Hypertrophic Cardiomyopathy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02329184
• Other study ID #: MYK-461-001
• Status: Completed
• Phase: Phase 1
• First received: December 19, 2014
• Last updated: September 12, 2016
• Start date: December 2014
• Est. completion date: April 2016

Study information

• Verified date: September 2016
• Source: MyoKardia, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: April 2016
• Est. primary completion date: March 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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)..

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Hypertrophic
• Hypertrophic Cardiomyopathy
• Hypertrophy

Intervention

Drug:
• MYK-461

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
MyoKardia, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Incidence of adverse events		28 Days	No
Secondary	Determination of pharmacokinetics parameters as measured by Cmax	maximum concentration (Cmax)	28 Days	No
Secondary	Determination of pharmacokinetics parameters as measured by Tmax	time of the maximum measured concentration (Tmax)	28 Days	No
Secondary	Determination of pharmacokinetics parameters as measured by AUC	area under the concentration-time curve (AUC)	28 Days	No
Secondary	Determination of pharmacokinetics parameters as measured by t1/2	half-life (t1/2)	28 Days	No