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August 11, 2026The Lancet

Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): health status analysis of a randomised, double-blind, placebo-controlled, phase 3 trial

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Why the study?

Available pharmacological treatments for obstructive hypertrophic cardiomyopathy are not disease-specific and often inadequate or poorly tolerated, leading investigators to assess the effect of mavacamten on patient health status.

Does mavacamten improve health status in adult patients with symptomatic obstructive hypertrophic cardiomyopathy?

Population

251 adults with symptomatic obstructive hypertrophic cardiomyopathy

Comparison

Mavacamten vs placebo

Design

Multicentre double-blind randomised placebo-controlled phase 3 trial

Follow-up

38 weeks

Key result

Mavacamten significantly improved health status in patients with symptomatic obstructive hypertrophic cardiomyopathy compared to placebo, with a mean KCCQ-OS score difference of +9.1 (P<0.0001).

Authors

JSJohn A. SpertusJFJennifer FinePEPerry Elliott

Discussion

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Overview

Mavacamten significantly improves symptoms, physical function, and quality of life in patients with symptomatic obstructive hypertrophic cardiomyopathy.

Key Points

  • This research aims to evaluate the impact of mavacamten on health status in patients with obstructive hypertrophic cardiomyopathy.
  • Conducted a randomized, double-blind, placebo-controlled trial with 251 adults enrolled.
  • Patients received mavacamten or placebo for 30 weeks, followed by an 8-week washout period.
  • Health status was measured using the Kansas City Cardiomyopathy Questionnaire at multiple time points.
  • At 30 weeks, mavacamten group showed a KCCQ-OS score increase of 14.9 compared to 5.4 in the placebo group (difference +9.1, 95% CI 5.5-12.8, p<0.0001).
  • 36% of mavacamten patients had a significant improvement (KCCQ-OS ≥20 points) vs 15% in placebo, yielding an absolute difference of 21%.
  • Improvements returned to baseline levels after stopping treatment.

Study Design

Type

RCT (n=251)

Blinding

double-blind

Randomization

1:1

Multicenter

Yes

Structured PICO

Does mavacamten improve health status in adult patients with symptomatic obstructive hypertrophic cardiomyopathy?

P
Population
251 adults (≥18 years) with symptomatic obstructive hypertrophic cardiomyopathy (gradient ≥50 mm Hg and NYHA class II-III) randomized to mavacamten or placebo for 30 weeks.
I
Intervention
Mavacamten for 30 weeks
C
Comparator
Placebo for 30 weeks
O
Outcome
Change from baseline to week 30 in Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary (OS) scorepatient reported

Main Result

Mean Difference: 9.1 (95% CI 5.5–12.8)

Absolute Event Rate: 14.9% vs 5.4%

Absolute Risk Reduction: 21%

Number Needed to Treat: 5

p-value: p=<0.0001

Mavacamten significantly improves symptoms, physical function, and quality of life in patients with symptomatic obstructive hypertrophic cardiomyopathy.

Cite This Study

Spertus et al. (2021) conducted an RCT in symptomatic obstructive hypertrophic cardiomyopathy (n=251). mavacamten vs. placebo was evaluated on Change from baseline to week 30 in KCCQ overall summary (OS) score (MD 9.1, 95% CI 5.5-12.8, p=<0.0001). Mavacamten significantly improved health status in patients with symptomatic obstructive hypertrophic cardiomyopathy compared to placebo, with a mean KCCQ-OS score difference of +9.1 (P<0.0001).

synapsesocial.com/papers/6a7b70264aa654994ddc2d4chttps://doi.org/10.1016/s0140-6736(21)00763-7
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial2020 · 1,266 citations
  2. 2Mavacamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy2024 · 82 citations
  3. 3Mavacamten monotherapy in obstructive hypertrophic cardiomyopathy: an integrated analysis of phase 3 clinical trials2026
  4. 4Study design and rationale of EXPLORER-CN: a phase III, randomised, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of mavacamten in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy2023 · 12 citations
  5. 5Mavacamten — a new disease-specific option for pharmacological treatment of symptomatic patients with hypertrophic cardiomyopathy2021 · 14 citations