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NCT Number: NCT06253221

A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Cardiomyopathy, Hypertrophic Aortic Stenosis, Subvalvular Aortic Valve Disease Aortic Valve Stenosis Cardiomyopathies Cardiovascular Diseases Heart Diseases Heart Valve Diseases

Age range

12 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Local Institution - 0020, Sydney, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of HCM
  • Presence of LVOT obstruction
  • Presence of symptoms

Exclusion criteria

  • Phenocopy diseases resulting in myocardial hypertrophy not related to sarcomere dysfunction
  • Evidence of LVEF <50% in prior 6 months
  • Planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery, HCM medication dose increase)

Other protocol-defined Inclusion/Exclusion criteria apply.

Treatment and study plan

Mavacamten

Drug

Specified dose on specified days

Other names: BMS-986427

Placebo

Drug

Specified dose on specified days

Primary outcomes

  1. Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient

    Time frame: At Week 28

Secondary outcomes

  1. Change from baseline in resting LVOT gradient

    Time frame: At Week 28

  2. Change from baseline in post-exercise peak LVOT gradient

    Time frame: At Week 28

  3. Change from baseline in maximal wall thickness

    Time frame: At Week 28

  4. Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')

    Time frame: At Week 28

  5. Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)

    Time frame: From baseline up to Week 28

  6. Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg

    Time frame: From baseline up to Week 28

  7. Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28

    Time frame: From baseline up to Week 28

  8. Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28

    Time frame: From baseline up to Week 28

  9. Number of participants with treatment-emergent adverse events (TEAEs)

    Time frame: Up to Week 218

  10. Number of participants with treatment-emergent serious adverse events (TESAEs)

    Time frame: Up to Week 218

  11. Change from baseline in electrocardiogram (ECG) (QT interval)

    Time frame: At Week 28

  12. Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%

    Time frame: Up to Week 200

  13. Number of participants with LVEF < 50%

    Time frame: Up to Week 200

  14. Trough observed plasma concentration (Ctrough)

    Time frame: Up to Week 200

  15. Post-dose plasma concentration of mavacamten

    Time frame: Up to Week 200

  16. Maximum observed concentration (Cmax)

    Time frame: Up to Week 200

  17. Area under the concentration-time curve (AUC)

    Time frame: Up to Week 200

  18. Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales

    Time frame: At Day 1 and Week 11

  19. Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain

    Time frame: At Week 28

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Official study title

A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Important dates

Study start
2024
Primary completion
2025
Study completion
2031
First posted
Feb 12, 2024
Registry last updated
Dec 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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