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

Primary Purpose

Dilated Cardiomyopathy

Status

Completed

Phase

Phase 1

Locations

Australia

Study Type

Interventional

Intervention

MYK-491 or placebo

About this trial

This is an interventional treatment trial for Dilated Cardiomyopathy

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

Nucleus Network

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Single oral dose of MYK-491

Single oral dose of placebo

Arm Description

single-dose, oral suspension

Outcomes

Primary Outcome Measures

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 Measures

Maximum observed plasma drug concentration (Cmax)

Maximum observed plasma concentration (Tmax)

Area under the plasma concentration-time curve (AUC)

First-order terminal elimination half-life (t1/2)

Mean retention time (MRT)

Full Information

NCT ID

NCT03062956

First Posted

February 16, 2017

Last Updated

March 21, 2018

Sponsor

MyoKardia, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03062956

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2018

Overall Recruitment Status

Completed

Study Start Date

January 16, 2017 (Actual)

Primary Completion Date

November 28, 2017 (Actual)

Study Completion Date

November 28, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

MyoKardia, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dilated Cardiomyopathy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Sequential Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Masking Description

Subjects will be randomized 6:2 to MYK-491:placebo within each cohort.

Allocation

Randomized

Enrollment

64 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Single oral dose of MYK-491

Arm Type

Experimental

Arm Description

single-dose, oral suspension

Arm Title

Single oral dose of placebo

Arm Type

Placebo Comparator

Arm Description

single-dose, oral suspension

Intervention Type

Drug

Intervention Name(s)

MYK-491 or placebo

Intervention Description

Oral suspension

Primary Outcome Measure Information:

Title

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

Time Frame

7 days

Secondary Outcome Measure Information:

Title

Maximum observed plasma drug concentration (Cmax)

Time Frame

7 days

Title

Maximum observed plasma concentration (Tmax)

Time Frame

7 days

Title

Area under the plasma concentration-time curve (AUC)

Time Frame

7 days

Title

First-order terminal elimination half-life (t1/2)

Time Frame

7 days

Title

Mean retention time (MRT)

Time Frame

7 days

Other Pre-specified Outcome Measures:

Title

The change from baseline in LVFS, LVEF, LVSV and SET by TTE

Time Frame

7 days

Title

SET while using photoplethysmography

Time Frame

7 days

Title

Relationship between MYK-491 plasma concentrations/PK parameters and PD parameters

Time Frame

7 days

Title

Plasma concentrations of metabolites of MYK-491 in plasma and urine

Time Frame

7 days

Title

Relationship between MYK-491 plasma concentration and QTc interval

Time Frame

7 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jason Lickliter, MD

Organizational Affiliation

Nucleus Network

Official's Role

Principal Investigator

Facility Information:

Facility Name

Nucleus Network

City

Melbourne

State/Province

Victoria

ZIP/Postal Code

3004

Country

Australia

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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