Hypertrophic Cardiomyopathy Symptom Release by BX1514M (Light-CARMIDO)

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.

Study Overview

• Status: Completed
• Conditions: Hypertrophic Cardiomyopathy
• Intervention / Treatment: Drug: Treatment BX1514M; Other: Walk distance test; Other: Exercise echocardiography; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 38
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Bordeaux, France
    • CHU de Bordeaux

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment group; 20 patients	Drug: Treatment BX1514M; 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.; Other: Walk distance test; Walk distance test during 6 minutes; Other: Exercise echocardiography
Placebo Comparator: Placebo group; 20 patients	Other: Walk distance test; Walk distance test during 6 minutes; Other: Exercise echocardiography; Drug: Placebo; Placebo will be given for 30 days

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: University Hospital, Bordeaux
• Investigators: Principal Investigator: Stéphane LAFITTE, Pr, University Hospital, Bordeaux

Study record dates

Study Major Dates

• Study Start (Actual): October 7, 2015
• Primary Completion (Actual): July 6, 2016
• Study Completion (Actual): July 6, 2016

Study Registration Dates

• First Submitted: October 27, 2015
• First Submitted That Met QC Criteria: October 28, 2015
• First Posted (Estimate): October 29, 2015

Study Record Updates

• Last Update Posted (Actual): February 10, 2022
• Last Update Submitted That Met QC Criteria: February 9, 2022
• Last Verified: February 1, 2017

More Information

Terms related to this study

• Keywords: Left ventricular obstruction
• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Pathological Conditions, Anatomical; Aortic Valve Disease; Heart Valve Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Hypertrophy; Cardiomyopathies; Cardiomyopathy, Hypertrophic
• Other Study ID Numbers: CHUBX 2015/14