Today, we will be looking into the latest research and clinical trials focused on hypertrophic cardiomyopathy in adults.

The following list has been carefully curated by evaluating the ongoing Phase 3 trials for hypertrophic cardiomyopathy, specifically targeting adults in the United States. Please note that the dates provided are approximate and subject to change. This compilation primarily features studies that have released updates within the past 12 months.

This series aims to offer a glimpse into upcoming innovations in the field and how the outcomes of these studies could potentially influence clinical guidelines related to the topic.

Without further ado, let us explore the list of Hypertrophic Cardiomyopathy Clinical Trials!

Quick View Table of Hypertrophic Cardiomyopathy Clinical Trials
Study TitlePhaseEnrollmentStart DateLast Update Posted
A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy (ODYSSEY-HCM)PHASE 3420 12/14/202210/26/2024
A Long-Term Safety Extension Study of Mavacamten in Adults Who Have Completed MAVERICK-HCM or EXPLORER-HCMPHASE 3282 9/27/20189/19/2024
A Study to Evaluate the Efficacy and Safety of Sotagliflozin in Symptomatic Obstructive and Non-obstructive Hypertrophic CardiomyopathyPHASE 3500 9/24/202410/18/2024
A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy (VALOR-HCM)PHASE 3112 7/6/20207/11/2024
Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM (FOREST-HCM)PHASE 3900 5/6/202112/4/2024
Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Metoprolol Succinate in Adults With Symptomatic oHCM (MAPLE-HCM)PHASE 3175 6/19/20239/20/2024
Efficacy and Safety Trial of Aficamten vs Placebo in Adults With Symptomatic oHCM (SEQUOIA-HCM) (SEQUOIA-HCM)PHASE 32822/1/202212/2/2024
Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM (ACACIA-HCM)PHASE 3420 8/30/202312/4/2024

Phase 3 Clinical Trials:

A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy (ODYSSEY-HCM)

Study Details | Source

  • Sponsor: Bristol-Myers Squibb
  • The purpose of this study is to evaluate the safety, tolerability, and efficacy of mavacamten compared with placebo in participants with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
  • Interventions:
    • Drug: Mavacamten
    • Other: Placebo
  • Primary Outcomes Measures:
    • Change from baseline in Kansas City Cardiomyopathy Questionnaire (23-item) Clinical Summary Score (KCCQ-23 CSS) at Week 48, Up to Week 48
    • Change from baseline in peak oxygen consumption (pVO2) at Week 48, Up to Week 48

A Long-Term Safety Extension Study of Mavacamten in Adults Who Have Completed MAVERICK-HCM or EXPLORER-HCM

Study Details | Source

  • Sponsor: Bristol-Myers Squibb
  • "Approximately 30 sites that enrolled participants in the MAVERICK-HCM (MYK-461-006) study in the United States (US) will initiate this study. Approximately 90 sites that enrolled participants in the EXPLORER-HCM (MYK-461-005) study in the US, Europe, and Israel will initiate this study. Note: Approximately 30 centers overlap between MAVERICK and EXPLORER."
  • Interventions:
    • Drug: Mavacamten
  • Primary Outcomes Measures:
    • Frequency and severity of treatment-emergent adverse events and serious adverse events, Time Frame - 252 weeks

A Study to Evaluate the Efficacy and Safety of Sotagliflozin in Symptomatic Obstructive and Non-obstructive Hypertrophic Cardiomyopathy

Study Details | Source

  • Sponsor: Lexicon Pharmaceuticals
  • The main purpose of the study is to determine the changes in symptoms and functional limitations in participants with symptomatic hypertrophic cardiomyopathy (HCM) treated with sotagliflozin as compared to placebo.
  • Interventions:
    • Drug: Sotagliflozin
    • Drug: Placebo
  • Primary Outcomes Measures:
    • Change from Baseline to Week 26 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS)
    • KCCQ is a 23-item, self-administered questionnaire that measure the participant's perception of their health status, including their heart failure (HF) symptoms, impact on physical and social function and how their HF impacts the quality of life. KCCQ quantifies 7 domains: physical limitations (6 items), symptom stability (1 item), symptom frequency (4 items), symptom burden (3 items), self-efficacy (2 items), quality of life (3 items) and social limitations (4 items). Scores are generated for each domain and scaled from 0 to 100, with 0 denoting the worst and 100 the best possible status. Higher scores reflect better health status. Time Frame - Baseline to Week 26

A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy (VALOR-HCM)

Study Details | Source

  • Sponsor: Bristol-Myers Squibb
  • This is a randomized, double-blind, placebo-controlled, multi-center study in the United States (U.S.) that will evaluate the effect of mavacamten treatment on reducing the number of septal reduction therapy (SRT) procedures performed in subjects with symptomatic obstructive hypertrophic cardiomyopathy (oHCM [also known as HOCM]) who are eligible for SRT based on ACCF/AHA 2011 and/or ESC 2014 guidelines.
  • Interventions:
    • Drug: Mavacamten
    • Drug: Placebo
  • Primary Outcomes Measures:
    • Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eligible at Week 16
    • Participants who decided to proceed with SRT or were eligible for SRT at week 16. Participants with missing assessments were classified as meeting the primary endpoint (did not improve).
    • SRT eligibility using the New York Heart Association Functional Class (NYHA) and left ventricular outflow tract (LVOT) assessments per the 2011 ACCF/AHA guideline clinical and hemodynamic criterion are below:
      • NYHA Class III or IV/ NYHA Class II with exertion-induced syncope/near syncope, AND
      • Dynamic LVOT gradient at rest or with provocation >= 50 mmHg.
    • NYHA Class II at week 16, the following rules will be applied:
      • NYHA Class II with history of exertional syncope/ syncope at baseline and at W16 is still NYHA Class II, they remain SRT eligible IF their maximal LVOT gradient is ≥ 50mmHg
      • NYHA Class III/IV at baseline and at W16 has improved to Class II, they are no longer SRT eligible UNLESS they have AE of exertional syncope or pre-syncope during the 16 weeks. Time Frame - Week 16.

Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM (FOREST-HCM)

Study Details | Source

  • Sponsor: Cytokinetics
  • The purpose of this study is to collect long-term safety and tolerability data for aficamten.
  • Interventions:
    • Drug: Aficamten (5 - 20 mg)
  • Primary Outcomes Measures:
    • Incidence of adverse events observed during dosing of aficamten in patients with HCM
    • Patient incidence of reported Adverse Events (AEs) , Time Frame - Baseline to End of study, up to 5 years

Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Metoprolol Succinate in Adults With Symptomatic oHCM (MAPLE-HCM)

Study Details | Source

  • Sponsor: Cytokinetics
  • The purpose of this study is to compare the efficacy and safety of aficamten (CK-3773274) compared with metoprolol succinate in adults with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction.
  • Interventions:
    • Drug: Aficamten (CK-3773274) (5 mg, 10 mg, 15 mg and 20 mg)
    • Drug: Placebo to match aficamten
    • Drug: Metoprolol succinate (50 mg, 100 mg, 150 mg and 200 mg)
    • Drug: Placebo to match metoprolol succinate
  • Primary Outcomes Measures:
    • Change in peak oxygen uptake (pVO2) by cardiopulmonary exercise testing (CPET)
    • Effect of aficamten compared with metoprolol succinate on exercise capacity in patients with symptomatic oHCM. Time Frame - Baseline to Week 24

Efficacy and Safety Trial of Aficamten vs Placebo in Adults With Symptomatic oHCM (SEQUOIA-HCM) (SEQUOIA-HCM)

Study Details | Source

  • Sponsor: Cytokinetics
  • The purpose of this study is to evaluate the efficacy and safety of aficamten (CK-3773274) in adults with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction.
  • Interventions:
    • Drug: CK-3773274 (5 mg, 10 mg, 15 mg and 20 mg)
    • Drug: Placebo to match CK-3773274
  • Primary Outcomes Measures:
    • Change in peak oxygen uptake (pVO2) by cardiopulmonary exercise testing (CPET)
    • Effect of CK-3773274 on exercise capacity in patients with symptomatic oHCM. Time Frame - Baseline to Week 24

Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM (ACACIA-HCM)

Study Details | Source

  • Sponsor: Cytokinetics
  • This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.
  • Interventions:
    • Drug: Aficamten
    • Drug: Placebo
  • Primary Outcomes Measures:
    • Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS)
    • Effect of aficamten compared with placebo on participant health status. Time Frame - Baseline to Week 36

Potential Guideline That May Be Affected Includes:

There you have it - a list of phase 3 Clinical Trials for Hypertrophic Cardiomyopathy as of January 2025. Stay tuned, for our next Guidelines+ Trials Rundown. Sign up for alerts and stay informed on the latest published guidelines and articles.

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