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

• Sponsor: MyoKardia, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02329184
• Collaborator: (none)
• Enrollment: 15
• Locations: 7
• Arm: 1
• Duration (Months): 16
• Patients Per Site: 2.1
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Hypertrophic Cardiomyopathy	Drug: MYK-461	Phase 1

Study Design

• Study Type: Interventional
• Actual Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-461 in Patient Volunteers With Hypertrophic Cardiomyopathy
• Study Start Date: Dec 1, 2014
• Actual Primary Completion Date: Mar 1, 2016
• Actual Study Completion Date: Apr 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: MYK-461	Drug: MYK-461

Outcome Measures

Primary Outcome Measures

1. Safety: Incidence of adverse events [28 Days]

Secondary Outcome Measures

1. Determination of pharmacokinetics parameters as measured by Cmax [28 Days]

   maximum concentration (Cmax)

2. Determination of pharmacokinetics parameters as measured by Tmax [28 Days]

   time of the maximum measured concentration (Tmax)

3. Determination of pharmacokinetics parameters as measured by AUC [28 Days]

   area under the concentration-time curve (AUC)

4. Determination of pharmacokinetics parameters as measured by t1/2 [28 Days]

   half-life (t1/2)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)..

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1		Scottsdale	Arizona	United States	85259
2		Cypress	California	United States	90630
3		Stanford	California	United States	94305
4		Boston	Massachusetts	United States	02111
5		Kalamazoo	Michigan	United States	49007
6		Durham	North Carolina	United States	27710
7		Nashville	Tennessee	United States	37232

Sponsors and Collaborators

• MyoKardia, Inc.

Investigators

• Study Director: Jonathan C Fox, MD, PhD, MyoKardia, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: MyoKardia, Inc.
• ClinicalTrials.gov Identifier: NCT02329184
• Other Study ID Numbers: MYK-461-001
• First Posted: Dec 31, 2014
• Last Update Posted: Sep 14, 2016
• Last Verified: Sep 1, 2016

• Additional relevant MeSH terms: Cardiomyopathies; Cardiomyopathy, Hypertrophic; Hypertrophy

Study Results

No Results Posted as of Sep 14, 2016