MYK-491 in Patients with heart failure and Reduced Ejection Fraction

• Research type

  Research Study

• Full 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

• IRAS ID

  254022

• Contact name

  Pierpaolo Pellicori

• Contact email

  pierpaolo.pellicori@glasgow.ac.uk

• Sponsor organisation

  MyoKardia, Inc.

• Eudract number

  2018-002239-11

• Duration of Study in the UK

  1 years, 10 months, 31 days

• Research summary

  Heart failure with reduced ejection fraction (HFrEF) and Dilated Cardiomyopathy (DCM) are major components within heart failure conditions

  DCM is a disease identified by the increase of the left heart chamber, the heart muscle becomes weak and insufficient blood is pumped around the body. The causes of DCM are multiple and could be due to insults to the myocardium or by innate genetic mutations. Organ compensation occurs by increasing heart rate, arterial tone and retaining salt and water. Eventually these responses become inadequate and lead to heart failure.

  Myocardia is developing a medicinal drug MYK-491 for the treatment of HFrEF and DCM.
  This is a Global phase Ib/IIa study, patients between 18 and 80 years old are expected to be enrolled across 18 research sites globally, with an overall participation of 24 months.

  The study is divided into 2 parts. Part 1/Single Dose (SAD), where the investigational medicine will be administered in single dose. Part 2/Multiple Dose (MAD), where the investigational medicine will be administered for 9 days in a row.
  The participants will only participate in Part 2/Multiple Dose, the second part of the study.

  In Part 2/Multiple Dose study consists of:
  -Screening period (28 days)
  -Dosing and Associated Assessments (21 days)
  -Follow-up (7 days)

  In Part 2 of the study initially all participants will receive placebo twice a day. Participants will then be randomised 3:1 to receive either active MYK 491 or placebo for 7 days.

  Only Part 2 will take place in the United Kingdom.

  The aim of this study is to understand safety and tolerability of MYK-491 in patients with DCM.

• REC name

  West of Scotland REC 1

• REC reference

  19/WS/0036

• Date of REC Opinion

  8 May 2019

• REC opinion

  Further Information Favourable Opinion