NCT02329184

Brief Summary

The purpose of this study is to establish initial safety, tolerability, pharmacokinetics and pharmacodynamics of MYK-461 in human subjects. This is a sequential group, single ascending (oral) dose study in NYHA Class I, II, or III patient volunteers aged 18-65 years.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Dec 2014

Geographic Reach
1 country

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2014

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

December 19, 2014

Completed
12 days until next milestone

First Posted

Study publicly available on registry

December 31, 2014

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2016

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2016

Completed
Last Updated

September 14, 2016

Status Verified

September 1, 2016

Enrollment Period

1.2 years

First QC Date

December 19, 2014

Last Update Submit

September 12, 2016

Conditions

Hypertrophic Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

  • Safety: Incidence of adverse events

    28 Days

Secondary Outcomes (4)

  • Determination of pharmacokinetics parameters as measured by Cmax

    28 Days

  • Determination of pharmacokinetics parameters as measured by Tmax

    28 Days

  • Determination of pharmacokinetics parameters as measured by AUC

    28 Days

  • Determination of pharmacokinetics parameters as measured by t1/2

    28 Days

Study Arms (1)

MYK-461

EXPERIMENTAL
Drug: MYK-461

Interventions

MYK-461DRUG
MYK-461

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosed with HCM
  • Normal left ventricular ejection fraction (LVEF)
  • NYHA class I, II or III

You may not qualify if:

  • Inherited metabolic disorders, myocardial infiltration or cardiofaciocutaneous syndrome (e.g., Noonan's, Fabry's).
  • History of clinically important atrial or ventricular arrhythmias
  • History of positive human immunodeficiency virus (HIV) test and/or seropositive for hepatitis C virus (HCV) or hepatitis B virus (HBV)..

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Unknown Facility

Scottsdale, Arizona, 85259, United States

Location

Unknown Facility

Cypress, California, 90630, United States

Location

Unknown Facility

Stanford, California, 94305, United States

Location

Unknown Facility

Boston, Massachusetts, 02111, United States

Location

Unknown Facility

Kalamazoo, Michigan, 49007, United States

Location

Unknown Facility

Durham, North Carolina, 27710, United States

Location

Unknown Facility

Nashville, Tennessee, 37232, United States

Location

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic

Interventions

MYK-461

Condition Hierarchy (Ancestors)

CardiomyopathiesHeart DiseasesCardiovascular DiseasesAortic Stenosis, SubvalvularAortic Valve StenosisAortic Valve DiseaseHeart Valve Diseases

Study Officials

  • Jonathan C Fox, MD, PhD

    MyoKardia, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 19, 2014

First Posted

December 31, 2014

Study Start

December 1, 2014

Primary Completion

March 1, 2016

Study Completion

April 1, 2016

Last Updated

September 14, 2016

Record last verified: 2016-09

Locations

US(7)