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

Dilated Cardiomyopathy Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03062956
• Other study ID #: MYK-491-001
• Status: Completed
• Phase: Phase 1
• First received: February 16, 2017
• Last updated: March 21, 2018
• Start date: January 16, 2017
• Est. completion date: November 28, 2017

Study information

• Verified date: March 2018
• Source: MyoKardia, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 64
• Est. completion date: November 28, 2017
• Est. primary completion date: November 28, 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Dilated
• Dilated Cardiomyopathy

Intervention

Drug:
• MYK-491 or placebo
Oral suspension

Locations

Country	Name	City	State
Australia	Nucleus Network	Melbourne	Victoria

Sponsors (1)

Lead Sponsor	Collaborator
MyoKardia, Inc.

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	The change from baseline in LVFS, LVEF, LVSV and SET by TTE		7 days
Other	SET while using photoplethysmography		7 days
Other	Relationship between MYK-491 plasma concentrations/PK parameters and PD parameters		7 days
Other	Plasma concentrations of metabolites of MYK-491 in plasma and urine		7 days
Other	Relationship between MYK-491 plasma concentration and QTc interval		7 days
Primary	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	Maximum observed plasma drug concentration (Cmax)		7 days
Secondary	Maximum observed plasma concentration (Tmax)		7 days
Secondary	Area under the plasma concentration-time curve (AUC)		7 days
Secondary	First-order terminal elimination half-life (t1/2)		7 days
Secondary	Mean retention time (MRT)		7 days