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Updated: Jul 25, 2025

Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model
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Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model

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Clinical Trials in Hypertrophic Cardiomyopathy Therapy: A Comprehensive Analysis of Trials Registered in Global

Huan Zhang1,2, Cheng Yu1, Yuanling Cheng1

  • 1Department of Cardiology, China Resources & Wisco General Hospital, Wuhan University of Science and Technology, Wuhan, People's Republic of China.

Drug Design, Development and Therapy
|June 28, 2023
PubMed

Insights

Clinical trials for hypertrophic cardiomyopathy (HCM) are increasing, but many are small and lack randomization or masking. Research is exploring new targets beyond myosin-7 to improve HCM therapies.

Area of Science:

  • Cardiology
  • Clinical Trials
  • Pharmacology

Background:

  • Disappointing results from cardiac myosin inhibitors for hypertrophic cardiomyopathy (HCM) have spurred rapid development of new therapeutic interventions.
  • ClinicalTrials.gov and the International Clinical Trials Registry Platform (ICTRP) are key resources for tracking these evolving HCM therapies.

Purpose of the Study:

  • To assess the characteristics of therapeutic interventions for hypertrophic cardiomyopathy (HCM) currently registered in major clinical trial databases.
  • To analyze trends in study design, drug classes, and molecular targets within recent HCM clinical trials.

Main Methods:

  • A cross-sectional, descriptive study analyzing 137 registered clinical trials for therapeutic interventions in hypertrophic cardiomyopathy (HCM).
  • Data were extracted from ClinicalTrials.gov and ICTRP, focusing on study design, participant numbers, drug types, and targeted pathways.

Main Results:

  • The majority of HCM trials (77.37%) were for treatment purposes, with many employing randomization (59.12%) and masking (45.26%).
  • A significant portion of trials (48.18%) enrolled fewer than 50 participants, and 27.74% were Phase 2 trials.
  • Myosin inhibitors, cardiovascular agents, and cation channel blockers were common drug classes, with myosin-7 and adrenergic receptors being frequently targeted pathways.

Conclusions:

  • The number of clinical trials for HCM therapeutic interventions has significantly increased.
  • Recent trials often feature small participant numbers and may lack robust designs like randomization or masking.
  • While myosin-7 is a focus, exploring novel molecular signaling pathways is crucial for future HCM treatment development.
Abstract

Keywords:
aminesclinical trialsmyosin inhibitorsmyosin-7nonobstructive hypertrophic cardiomyopathyobstructive hypertrophic cardiomyopathy

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