Phase 2a study of danicamtiv in MYH7-DCM or TTN-DCM patients

  • Research type

    Research Study

  • Full title

    An Open-Label Exploratory Study of the Safety and Preliminary Efficacy of Danicamtiv in Stable Ambulatory Patients with Primary Dilated Cardiomyopathy due either MYH7 or TTN variants.



  • Contact name

    James Ware

  • Contact email

  • Sponsor organisation

    MyoKardia Inc.

  • Eudract number


  • Identifier


  • Duration of Study in the UK

    1 years, 2 months, 0 days

  • Research summary

    Heart failure is a long-term illness where the heart has been damaged and does not pump blood through the body as well as it should. Symptoms of heart failure can include shortness of breath (dyspnoea), lack of energy (fatigue), build-up of fluid (oedema), chronic coughing/wheezing and confusion.

    Primary Dilated Cardiomyopathy (DCM) is a condition in which the heart becomes stretched and weakened due to changes in the structure or function of the heart muscle, which in turn means the heart cannot pump blood effectively. This is one of the leading causes of heart failure.

    DCM may affect up to 1in250 individuals worldwide, and in some 2-3% of cases, the condition is caused by a variant in a single gene called MYH7.

    Although current treatments are effective and improve clinical outcomes, long lasting and well tolerated therapies that directly target the weakened heart muscle and address the underlying cause are lacking.

    MYK-491 is being developed by Myokardia Inc. to improve the pumping function of the heart muscle for the treatment of heart failure with reduced ejection fraction (HFrEF) and DCM, which together represent about 40% of heart failure.

    This is a Phase IIa Study. Approximately 12 participants between 18 and 80 years old are expected to be enrolled across 12 research sites globally, with an overall participation of 4-11 weeks.

    The study will consist of 2 sequential, open-label, treatment periods where all participants will receive the active study medicine, MYK-491 tablets to be taken twice a day. Both treatment periods 1 and 2 will each last 5-8 days.

    Treatment Period 1: MYK-491 25mg twice a day

    Treatment Period 2: MYK-491 50mg OR 10mg twice a day depending on the results from TP1

    The aim of the study is to understand the safety and tolerability of the study medicine MYK-491 in participants with Primary DCM due to MYH7 variant.

  • REC name

    London - Central Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    15 Jun 2020

  • REC opinion

    Further Information Favourable Opinion

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