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Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model
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Current State of Hypertrophic Cardiomyopathy Clinical Trials

Hussein H Khachfe1, Hamza A Salhab1, Mohamad Y Fares1

  • 1Faculty of Medicine, American University of Beirut, Beirut, Lebanon; Neuroscience Research Center, Faculty of Medical Sciences, Lebanese University, Beirut, Lebanon.

Global Heart
|August 28, 2019
PubMed

Insights

Clinical trials for hypertrophic cardiomyopathy (HCM) are limited, with few published results and a lack of geographic diversity. Improving HCM trial management is crucial for developing new therapies.

Area of Science:

  • Cardiovascular Medicine
  • Genetics
  • Clinical Research

Background:

  • Hypertrophic cardiomyopathy (HCM) is a prevalent genetic disorder with significant global health implications.
  • Existing therapeutic management strategies for HCM require enhancement.

Purpose of the Study:

  • To analyze the landscape of clinical trials for hypertrophic cardiomyopathy.
  • To identify limitations in current HCM trial designs and execution.
  • To propose improvements for future HCM therapeutic development.

Main Methods:

  • A comprehensive search of ClinicalTrials.gov was conducted in January 2019 for all HCM therapeutic and interventional trials.
  • Data collected included trial phase, duration, location, sample size, interventions, and publication status.
  • Analysis focused on trial characteristics and outcomes to identify trends and shortcomings.

Main Results:

  • Sixty-three trials met the inclusion criteria, with an average duration of approximately 3 years.
  • North America was the primary location for trials (50%), and 44% were in early phases (I/II).
  • A low publication rate (14 publications) was observed across all studied trials.

Conclusions:

  • The current body of HCM clinical trials is insufficient, lacking geographic diversity and robust publication of results.
  • Addressing these limitations is essential for advancing effective therapeutic options for hypertrophic cardiomyopathy.
  • Optimizing the management and reporting of HCM clinical trials is critical for future progress.
Abstract

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