Hypertrophic Cardiomyopathy Symptom Release by BX1514M

Study Overview
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Brief Summary
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Hypertrophic cardiomyopathy (HCM) is the most frequent genetic cardiac disease characterized by an asymmetric hypertrophic. In two third of patients, an obstruction to blood ejection is observed within the left ventricle which is named left ventricular outflow tract obstruction (LVOTO). This phenomenon can occur at rest or during exercise and is associated with symptoms such as dyspnea, dizziness or chest pain that can significantly limit day life adaptation. To now, medical or interventional treatments such as betablocacker, calcium blockers or septal alcoholisation or surgery present with a limited efficiency. Recent studies from investigators group revealed new concepts about the role of venous return to the LVOTO. Therefore the investigators hypothesis that BX1514M generating a venous vascular constriction, could improve symptoms of HCM patients by reducing LVOTO.

Conditions
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Hypertrophic Cardiomyopathy

Intervention / Treatment
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* Drug: Treatment BX1514M
* Drug: Placebo
* Other: Walk distance test
* Other: Exercise echocardiography


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a typical or atypical hypertrophic cardiomyopathy (HCM) confirmed by imaging modalities (echo and MRI) and/or genetic tests or a relative history of HCM Left ventricular obstruction during exercise on treadmill (above 50 mmHg) Positive response to leg lifting test (obstruction reduction above 20 mmHg) at rest or in early recovery phase Daily symptoms as shortness of breath during exercise (NYHA2-3), non coronary chest pain, dizziness Correct ultrasound windows quality Sinus rhythm Optimal medical treatment For women, pregnancy test or contraception Written consent form obtained Exclusion Criteria: Previous treatment by BX1514M Extra-cardiac pathology with life expectancy below than 1 year No capability of consent form written Pregnancy women Secondary hypertension hypertrophy, secondary valvular disease hypertrophy Permanent atrial fibrillation Severe ventricular arrhythmia without Implantable Cardioverter Defibrillator (ICD) Severe coronary disease Severe non stabilized hypertension Severe cardiopathy (ejection fraction below 40% or demonstrated elevated end diastolic pressure or pulmonary pressure above 60 mmHg) Bradycardia Narrow angle glaucoma Vascular prethrombotic diseases Vascular spams Thyrotoxicosis Pheochromocytoma Severe renal failure (<30ml/mn) Patients at risk of urinary retention secondary to prostatic severe disease Raynaud's disease Non selective Monoamine Oxidase Inhibitors (MAOIs), iproniazid, nialamide or ephedrine, methylphenidate, phenylephrine, pseudoephedrine or digitalic Procedures modifying the peripheral venous return Participation to other research protocol

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment group<br>20 patients | Drug: Treatment BX1514M<br>* X mg x 3 /day of BX1514M for 15 days. Dosage will be then adapted, depending on symptoms release and walk distance test results for another 15 days period.<br>Other: Walk distance test<br>* Walk distance test during 6 minutes<br>Other: Exercise echocardiography<br> <br> |
| Placebo Comparator: Placebo group<br>20 patients | Drug: Placebo<br>* Placebo will be given for 30 days<br>Other: Walk distance test<br>* Walk distance test during 6 minutes<br>Other: Exercise echocardiography<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline at day 15 of covered distance during the 6 minutes walk distance test (6MWT) | In meters | Day 15 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Covered distance during the 6 minutes walk distance test (6MWT) | In meters | Day 30 | 
| Exercise echocardiography with measures of blood pressures in the rest and in the effort |  | Day 1, day 15, day 30 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Left ventricular obstruction