Farrant, JP, Schmitt, M, Reid, AB, Garratt, CJ, Newman, WG, Malhotra, A, Beynon, R, Mahmod, M, Raman, B, Cooper, RM, Dawson, D, Green, T, Prasad, SK, Singh, A, Dodd, S, Watkins, H, Neubauer, S and Miller, CA (2024) Considerations for drug trials in hypertrophic cardiomyopathy. ESC Heart Failure.

Preview

Text Considerations for drug trials in hypertrophic cardiomyopathy.pdf - Published Version Available under License Creative Commons Attribution. Download (776kB) | Preview

Abstract

Hypertrophic cardiomyopathy (HCM) is a heterogeneous condition with potentially serious manifestations. Management has traditionally comprised therapies to palliate symptoms and implantable cardioverter-defibrillators to prevent sudden cardiac death. The need for disease-modifying therapies has been recognized for decades. More recently, an increasing number of novel and repurposed therapies hypothesized to target HCM disease pathways have been evaluated, culminating in the recent regulatory approval of mavacamten, a novel oral myosin inhibitor. HCM poses several unique challenges for clinical trials, which are important to recognize when designing trials and interpreting findings. This manuscript discusses the key considerations in the context of recent and ongoing randomized trials, including the roles of genotype, phenotype and symptom status in patient selection, the evidence base for clinical and mechanistic outcome measurements, trial duration and sample size.

Item Type:	Article
Uncontrolled Keywords:	1102 Cardiorespiratory Medicine and Haematology
Subjects:	R Medicine > RC Internal medicine > RC1200 Sports Medicine R Medicine > RS Pharmacy and materia medica
Divisions:	Sport and Exercise Sciences
Publisher:	Wiley
SWORD Depositor:	A Symplectic
Date Deposited:	06 Nov 2024 10:41
Last Modified:	06 Nov 2024 10:45
DOI or ID number:	10.1002/ehf2.15138
URI:	https://researchonline.ljmu.ac.uk/id/eprint/24683

View Item