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Exercise Capacity in Patients With Obstructive Hypertrophic Cardiomyopathy: SEQUOIA-HCM Baseline Characteristics and
Caroline J Coats1, Martin S Maron2, Theodore P Abraham3
1School of Cardiovascular and Metabolic Health, University of Glasgow, Glasgow, United Kingdom.
Insights
Obstructive hypertrophic cardiomyopathy (oHCM) patients face quality of life issues despite current treatments. The SEQUOIA-HCM trial investigates aficamten to improve exercise capacity and symptoms in oHCM.
Area of Science:
- Cardiology
- Clinical Trials
- Pharmacology
Background:
- Obstructive hypertrophic cardiomyopathy (oHCM) increases risks of serious cardiac events.
- Despite contemporary management, oHCM patients experience reduced quality of life and persistent symptoms.
Purpose of the Study:
- To evaluate the efficacy of aficamten in treating obstructive hypertrophic cardiomyopathy.
- To assess the impact of aficamten on functional capacity and symptoms in oHCM patients.
Main Methods:
- The SEQUOIA-HCM trial is a Phase 3, randomized, placebo-controlled study.
- Enrolled 282 ethnically diverse patients with oHCM receiving standard-of-care.
- Assessed aficamten versus placebo over 24 weeks for functional capacity and symptoms.
Main Results:
- Baseline characteristics represent a diverse oHCM population with significant symptom burden.
- The trial is fully enrolled, with data collection ongoing.
Conclusions:
- The SEQUOIA-HCM trial design provides a model for future oHCM therapy evaluations.
- Aficamten is being investigated as a potential therapy to alleviate oHCM burden.
Abstract:
Patients with obstructive hypertrophic cardiomyopathy (oHCM) have increased risk of arrhythmia, stroke, heart failure, and sudden death. Contemporary management of oHCM has decreased annual hospitalization and mortality rates, yet patients have worsening health-related quality of life due to impaired exercise capacity and persistent residual symptoms. Here we consider the design of clinical trials evaluating potential oHCM therapies in the context of SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM). This large, phase 3 trial is now fully enrolled (N = 282). Baseline characteristics reflect an ethnically diverse population with characteristics typical of patients encountered clinically with substantial functional and symptom burden. The study will assess the effect of aficamten vs placebo, in addition to standard-of-care medications, on functional capacity and symptoms over 24 weeks. Future clinical trials could model the approach in SEQUOIA-HCM to evaluate the effect of potential therapies on the burden of oHCM. (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM [SEQUOIA-HCM]; NCT05186818).
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