MYK 461-001 Safety and tolerability of MYK 461 in HV & Patients
Research type
Research Study
Full title
Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-461 in Patient Volunteers with Hypertrophic Cardiomyopathy and Healthy Volunteers: A First in Human Study
IRAS ID
168846
Contact name
James Ritter
Contact email
Sponsor organisation
MyoKardia Inc
Eudract number
2014-005063-33
Duration of Study in the UK
0 years, 8 months, 12 days
Research summary
MYK-461 is a drug that may be useful in the treatment of hypertrophic cardiomyopathy (HCM), a genetic disorder of the heart muscle where this becomes thickened, beats too forcefully and/or is stiff and does not relax properly. MYK-461 is meant to reduce the force of contraction and promote better relaxation of the abnormal heart muscle.
The purpose of this study is to examine the safety of MYK-461 and, in particular, its effects on the heart measured non-invasively with echocardiography, its tolerability and the way the body handles (absorbs, distributes, breaks down and excretes) this drug. In this study various increasing single oral doses of MYK-461 will be investigated in patients with HCM and also in healthy volunteers. This study will be the first time MYK-461 is given to clinically stable patient volunteers with HCM and to healthy human volunteers and will provide information to facilitate development of this drug for use in HCM patients.
This study will involve outpatient screening followed by a single admission of six days and five nights resident in the drug research unit, plus several outpatient visits.REC name
London - Bloomsbury Research Ethics Committee
REC reference
15/LO/0017
Date of REC Opinion
12 Mar 2015
REC opinion
Further Information Favourable Opinion