A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491

Phase 1

Completed

• Conditions: Dilated Cardiomyopathy
• Interventions: Drug: MYK-491 or placebo

• Registration Number: NCT03062956
• Lead Sponsor: MyoKardia, Inc.

Brief Summary

Up to 72 healthy volunteers will be given a single dose of MYK-491 or placebo and be monitored for safety and tolerability over a 7 day period.

Detailed Description

Up to 72 healthy volunteers will be given a single dose of MYK-491 or placebo and be monitored for safety and tolerability over a 7 day period. After the 28 day screening period, the eligible subject will be admitted to the clinical site and will receive a single dose of study drug or placebo. Subjects will be confined to the clinical site for five days (Day -1 to Day 4) and will return to the clinic on Day 7 for a safety follow-up.

Study Timeline

• Study Start: 2017-01-16
• Study First Submitted: 2017-02-16
• Study First Submitted QC: 2017-02-20
• Study First Posted: 2017-02-24
• Primary Completion: 2017-11-28
• Study Completion: 2017-11-28
• Status Verified: 2018-03
• Last Update Submitted: 2018-03-21
• Last Update Posted: 2018-03-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 64

Inclusion Criteria

• Weight between 60 and 90 kg inclusive
• Resting heart rate of < 80 beats per minute
• Documented LVEF greater than or equal to 55% during Screening
• Normal electrocardiogram (ECG) at Screening
• Normal acoustic windows on transthoracic echocardiograms at Screening
• All safety laboratory parameters within normal limits at Screening
• History or evidence of another clinically significant disorder, in the opinion of the investigator.

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Exclusion Criteria

• Active infection
• History of coronary artery disease
• History of malignancy with the exception of in situ cervical cancer more than 5 years prior to Screening or surgically-excised non-melanomatous skin cancers more than 2 years prior to Screening
• Positive serology tests at screening
• Current use of tobacco or nicotine-containing products exceeding 10 per day.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Single oral dose of MYK-491	MYK-491 or placebo	single-dose, oral suspension
Single oral dose of placebo	MYK-491 or placebo	single-dose, oral suspension

Primary Outcome Measures

Name	Time	Method
Safety and tolerability assessments will include treatment emergent AEs and SAEs, ECG recordings, vital signs, hs-troponin 1 concentrations, laboratory abnormalities and physical exam abnormalities	7 days

Secondary Outcome Measures

Name	Time	Method
Maximum observed plasma drug concentration (Cmax)	7 days
Maximum observed plasma concentration (Tmax)	7 days
Area under the plasma concentration-time curve (AUC)	7 days
First-order terminal elimination half-life (t1/2)	7 days
Mean retention time (MRT)	7 days

Trial Locations

Locations (1)

Nucleus Network; 🇦🇺 Melbourne, Victoria, Australia