Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of MYK-461 in Healthy Volunteers

A Phase 1, Double-blind, Randomized, Placebo-controlled, Ascending Multiple Oral Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of MYK-461 in Healthy Volunteers

The purpose of this study is to establish safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of MYK-461 in human subjects. This is a randomized, double-blind, placebo-controlled, sequential group, multiple ascending (oral tablet) dose study in healthy volunteers aged 18-55 years.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: Placebo; Drug: MYK-461

• Study Type: Interventional
• Enrollment (Actual): 60
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia
      • Nucleus Network

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Normal body mass index (BMI)
• Normal LVEF
• Normal electrocardiogram (ECG)
• Females must be non pregnant, non lactating and, if sexually active, be using an acceptable birth control method from the time of first dose through 3 months after the last dose of study drug

Exclusion Criteria:

• Any structural abnormalities on echocardiography
• Positive results of HIV test and/or seropositive for HCV or HBV

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: TRIPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MYK-461; Oral Tablet x 28 days	Drug: MYK-461
Placebo Comparator: Placebo; Oral Tablet x 28 days	Drug: Placebo; Placebo comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety as measured by the incidence of adverse events	Incidence of adverse events	63 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Determination of pharmacokinetic parameters	maximum concentration (Cmax)	63 days
Determination of pharmacokinetic parameters	time of the maximum measured concentration (Tmax)	63 days
Determination of pharmacokinetic parameters	area under the concentration time curve (AUC)	63 days
Determination of pharmacokinetic parameters	half-life (t1/2)	63 days
Characterize pharmacodynamic parameters	echocardiogram assessment	63 days
Characterize pharmacodynamic parameters	peak oxygen consumption	63 days

Collaborators and Investigators

• Sponsor: MyoKardia, Inc.
• Investigators: Study Director: Michael Grimm, MD, MyoKardia, Inc.

Study record dates

Study Major Dates

• Study Start: August 1, 2015
• Primary Completion (Actual): July 1, 2016
• Study Completion (Actual): October 1, 2016

Study Registration Dates

• First Submitted: June 17, 2015
• First Submitted That Met QC Criteria: June 22, 2015
• First Posted (Estimate): June 24, 2015

Study Record Updates

• Last Update Posted (Actual): March 22, 2017
• Last Update Submitted That Met QC Criteria: March 21, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Other Study ID Numbers: MYK461-003