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Study Evaluating the Safety, Tolerability and Preliminary Pharmacokinetics and Pharmacodynamics of MYK-491

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03447990
Recruitment Status : Recruiting
First Posted : February 27, 2018
Last Update Posted : August 30, 2019
Information provided by (Responsible Party):
MyoKardia, Inc.

Brief Summary:
The purpose of this Phase 1b/2a study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of MYK-491 in patients with DCM and stable heart failure.

Condition or disease Intervention/treatment Phase
Dilated Cardiomyopathy Heart Failure With Reduced Ejection Fraction Drug: MYK-491 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 56 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

This is a a two part study. The first part is a randomized, crossover, double-blind, placebo-controlled, two cohort, sequential ascending single dose study. All patients will receive placebo and active doses of MYK-491.

The second part is a randomized, parallel, double-blind, placebo-controlled, sequential ascending multiple dose study. All patients will receive placebo and/or active doses of MYK-491.

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Double-blind, Placebo-controlled, Two-Part, Adaptive Design Study of Safety, Tolerability, Preliminary Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Oral Doses of MYK-491 in Patients With Stable Heart Failure With Reduced Ejection Fraction
Actual Study Start Date : February 6, 2018
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Arm Intervention/treatment
Part 1/SAD
Crossover, Single ascending dose of MYK-491/placebo
Drug: MYK-491
Single Ascending Dose of MYK-491/placebo

Part 2/MAD
Parallel, Multiple ascending dose of MYK-491/placebo
Drug: MYK-491
Multiple Ascending Dose of MYK-491/placebo

Primary Outcome Measures :
  1. Frequency and severity of treatment-emergent adverse events and serious adverse events. [ Time Frame: Up to 49 days ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Has stable chronic heart failure with reduced ejection fraction
  • Has adequate acoustic windows for echocardiography

Key Exclusion Criteria:

  • Any significant structural cardiac abnormalities on Screening TTE
  • At Screening, symptomatic hypotension or hypertension or bradycardia.
  • Routinely scheduled outpatient intravenous (IV) infusions for heart failure (e.g., inotropes, vasodilators [e.g., nesiritide], diuretics) or routinely scheduled ultrafiltration.
  • Presence of protocol specified laboratory abnormalities at Screening.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03447990

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Contact: MyoKardia Medical Affairs 650-741-0900

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United States, Florida
Jacksonville Center for Clinical Research Recruiting
Jacksonville, Florida, United States, 32216
Contact: Richard Smith    904-732-9255      
Principal Investigator: Michael Koren         
United States, Minnesota
Prism Reseach Recruiting
Saint Paul, Minnesota, United States, 55102
Principal Investigator: Les Forgosh         
United States, Missouri
St. Louis Heart and Vascular Cardiology Recruiting
Saint Louis, Missouri, United States, 63136
Principal Investigator: Harvey Serota         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Principal Investigator: Ravi Karra         
United States, Ohio
Ohio State University Medical Center Recruiting
Columbus, Ohio, United States, 43210
Principal Investigator: Ray Hershberger         
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Principal Investigator: Samuel Camacho         
United States, Pennsylvania
University of Pennsylvania Heart and Vascular Center Recruiting
Philadelphia, Pennsylvania, United States, 19104
Principal Investigator: Anjali Owens         
United States, Tennessee
Tennessee Center for Clinical Trials Recruiting
Tullahoma, Tennessee, United States, 37388
Principal Investigator: Dinesh Gupta         
Charite Research Organization Recruiting
Berlin, Germany, 10117
Principal Investigator: Frank Wagner         
Groningen UMC Recruiting
Groningen, Netherlands, 9713 GZ
Principal Investigator: Adriaan Voors         
D&A Research Recruiting
Sneek, Netherlands, 8601
Principal Investigator: Henk Swart         
Karolinska University Hospital Recruiting
Stockholm, Sweden, 17164
Principal Investigator: Lars Lund         
Sponsors and Collaborators
MyoKardia, Inc.

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Responsible Party: MyoKardia, Inc. Identifier: NCT03447990     History of Changes
Other Study ID Numbers: MYK-491-003
First Posted: February 27, 2018    Key Record Dates
Last Update Posted: August 30, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Heart Failure
Cardiomyopathy, Dilated
Heart Diseases
Cardiovascular Diseases
Antifungal Agents
Anti-Infective Agents