Clinical trial: Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy (NCT03470545)

Eligibility criteria:
Key Inclusion Criteria:

          -  Age 18 and greater, body weight ≥ 45kg

          -  Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)

          -  Diagnosed with oHCM consistent with current American College of Cardiology
             Foundation/American Heart Association and European Society of Cardiology guidelines
             and satisfy both criteria:

          -  Has documented left ventricular ejection fraction (LVEF) ≥55%

          -  NYHA Class II or III

          -  Has documented oxygen saturation at rest ≥90% at Screening

          -  Is able to perform an upright CPET and has a respiratory exchange ratio (RER) ≥1.0 at
             Screening per central reading

        Key Exclusion 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
             life-threatening ventricular arrhythmia within 6 months prior to Screening

          -  Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at
             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 disopyramide or ranolazine

          -  Treatment (within 14 days prior to Screening) or planned treatment during the study
             with a combination of β-blockers and calcium channel blockers

          -  LVOT gradient with Valsalva maneuver <30 mmHg at Screening

          -  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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.


Patient description (medical language): A 23-year-old man comes to the emergency department following an episode of syncope.  He was working out when he felt dizzy and passed out without head injury.  He has had 3 other episodes of light-headedness over the last year, all happening during physical activity. He never had this experience while resting. He has no other medical conditions.  The patient does not use tobacco, alcohol, or illicit drugs.  His father died suddenly at age 35.  Vital signs are within normal limits.  On physical examination, the patient has a harsh systolic murmur.  The lungs are clear with no peripheral edema. Echocardiography shows asymmetric interventricular septal hypertrophy.

Patient description (patient's own words): I'm 23, and I got admitted to the ER because I fainted all of a sudden. I was in the gym, working out, when all of a sudden, I just got dizzy and passed out. Hopefully, I didn't hit my head. It's not my first time having that kind of dizziness at the gym, never when I'm chilling at home tho. I don't have any special medical conditions and I'm not even smoking, drinking or doing drugs! My dad died suddenly when he was 35, so I'm kind of scared. My vitals were normal while at the ER and they did a physical exam and they told me my heart sounds abnormal. My lungs are ok, but I had to do an echography of my heart, and they told me it is bigger than average.

Does the patient match the trial criteria? Entailment