Clinical trial: Role of Perhexiline in Hypertrophic Cardiomyopathy (NCT04426578)

Eligibility criteria:
Inclusion Criteria:

          1. Left Ventricular Ejection Fraction (LVEF) =/> 55% by echocardiography or CMR during
             the screening period or within 6 months prior to study entry

          2. Current / prior symptom(s) of HCM (New York Heart Association [NYHA] functional class
             II or class III, Canadian Cardiovascular Society [CCS] grade II or grade III) and
             requiring treatment with ß-blockers and /or non-dihydropyridine calcium antagonists
             and / or disopyramide for at least 30 days prior to study entry

          3. Structural heart disease as evidenced by interventricular septal thickness of (= 15
             mm) on echocardiography or CMR in the absence of abnormal loading conditions

          4. Elevated N terminal pro-brain natriuretic peptide (NT-proBNP), >125 pg/ml

        Exclusion Criteria:

          1. Any prior echocardiographic or CMR measurement of LVEF <55%

          2. Current acute decompensated heart failure requiring hospitalisation and / or augmented
             medical therapy

          3. Cardiac surgery or catheter-based septal reduction therapy planned or having occurred
             within the past 1 year

          4. Patients with a non-CMR conditional pacemaker / implantable cardioverter-defibrillator
             device

          5. History of a known chronic liver disease, peripheral neuropathy, recurrent
             hypoglycemia

          6. Serum bilirubin, alanine aminotransferase, aspartate aminotransferase, alkaline
             phosphatase, or lactate dehydrogenase > 2.0 times upper limit of normal

          7. Previous adverse reaction to perhexiline at therapeutic plasma levels of the drug

          8. Concomitant use of amiodarone, ranolazine or trimetazidine

          9. Life-threatening or uncontrolled dysrhythmia

         10. Contraindications to CMR, gadolinium, adenosine
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? 1