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

NCT ID: NCT03062956

Last Updated: 2018-03-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 64 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-16
• Study Completion Date: 2017-11-28

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.

Conditions

Dilated Cardiomyopathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Single oral dose of MYK-491

single-dose, oral suspension

Group Type: EXPERIMENTAL

MYK-491 or placebo

Intervention Type: DRUG

Oral suspension

Single oral dose of placebo

single-dose, oral suspension

Group Type: PLACEBO_COMPARATOR

MYK-491 or placebo

Intervention Type: DRUG

Oral suspension

Interventions

MYK-491 or placebo

Oral suspension

Intervention Type: DRUG

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

MyoKardia, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jason Lickliter, MD

Role: PRINCIPAL_INVESTIGATOR

Nucleus Network

Locations

Nucleus Network

Melbourne, Victoria, Australia

Countries

Australia

Other Identifiers

MYK-491-001

Identifier Type: -

Identifier Source: org_study_id