Background: Hypertrophic cardiomyopathy (HCM) is a genetic disorder with a very large global burden for which more therapeutic management regimens are required.

Objectives: In this study, the authors explore HCM-related clinical trials, determine the shortcomings leading to the lack of development of new HCM therapies, and attempt to shed light on potential areas for improvement.

Methods: In January 2019, the authors completed a search on ClinicalTrials.gov for all therapeutic and interventional clinical trials involving HCM, without any limits for location or date. Information on trial characteristics such as phase, start and end dates, sample size, experimental intervention, publications, study design, selection criteria, and results were collected and analyzed.

Results: Sixty-three trials met the selection criteria. The average trial duration across phases was around 3 years. Around one-half of the trials were conducted in North America (United States and Canada) and 44% of the trials were in their early phases (I and II). Approximately one-third of the trials were completed. Only 14 publications were produced from all the clinical trials studied.

Conclusions: The study revealed a low number of trials, lack of geographic diversity, and scarcity of published results concerning HCM clinical trials. Proper management of HCM trials is of vast importance to achieve effective therapies.

Highlights

• Only 63 intervention clinical trials have been conducted for hypertrophic cardiomyopathy since it was discovered.
• Most clinical trials have been phase II trials.
• Only 14 publications have been produced from all interventional hypertrophic cardiomyopathy clinical trials.
• All the clinical trials were conducted in only 15 countries.