Brief Title
A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491
Official Title
Randomized, Placebo-Controlled Study of Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-491 in Healthy Adult Volunteers
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 Phase
Phase 1
Study Type
Interventional
Primary Outcome
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
Secondary Outcome
Maximum observed plasma drug concentration (Cmax)
Condition
Dilated Cardiomyopathy
Intervention
MYK-491 or placebo
Study Arms / Comparison Groups
Single oral dose of MYK-491
Description: single-dose, oral suspension
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
64
Start Date
January 16, 2017
Completion Date
November 28, 2017
Primary Completion Date
November 28, 2017
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.
Gender
All
Ages
18 Years - 55 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Contacts
Jason Lickliter, MD, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT03062956
Organization ID
MYK-491-001
Responsible Party
Sponsor
Study Sponsor
MyoKardia, Inc.
Study Sponsor
Jason Lickliter, MD, Principal Investigator, Nucleus Network
Verification Date
March 2018