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

JRCT ID: jRCT2031220178

Registered date:02/07/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Obstructive Hypertrophic Cardiomyopathy
• Date of first enrollment: 19/08/2022
• Target sample size: 30
• Study type: Interventional

Intervention(s)

Single Group Assignment: Mavacamten
Participants will receive treatment with 1, 2.5, 5, 10, 1mg dose once daily.

Outcome(s)

• Primary Outcome: Change from Baseline to Week 30 in post exercise left ventricular outflow tract (LVOT) peak gradient as determined by Doppler echocardiography

Secondary Outcome

1.Change from Baseline to Week 30 in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS).
2.Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) functional class from baseline to Week 30.
3.Change from baseline to Week 30 in N-terminal pro b-type natriuretic peptide (NT-proBNP).
4.Change from baseline to Week 30 in cardiac troponins.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Age 18 and greater, body weight >- 35kg
- Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
- Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation
Society guidelines
- Has documented left ventricular ejection fraction (LVEF) >-60% NYHA Class II or III

Exclude criteria

- Known infiltrative or storage disorder causing cardiac hypertrophy that
mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome
with LV hypertrophy
- History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
- History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for lifethreatening ventricular arrhythmia within 6 months prior to Screening
- Paroxysmal atrial fibrillation with atrial fibrillation present at the time of Screening.
- Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
- Treatment (within 14 days prior to Screening) or planned treatment during
the study with cibenzoline, disopyramide or ranolazine
- Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
- Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the
study
- ICD placement within 2 months prior to Screening or planned ICD placement during the study
- Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation procedures, or completion
- Prior treatment with cardiotoxic agents such as doxorubicin or similar

Related Information

• Primary Sponsor: Florea Victoria
• Secondary ID(s): NCT05414175