Bisoprolol in DMD Early Cardiomyopathy

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Cardiomyopathy, Dilated; Duchenne Muscular Dystrophy

Treatments: Drug: Bisoprolol Fumarate

Study type: Interventional

Funder types: Other

Identifiers

NCT03779646

PekingUMCH-DMD

Details and patient eligibility

About

This study aimed to use cardiac magnetic resonance imaging (CMR) to evaluate the efficacy and safety of bisoprolol therapy for boys with Duchenne muscular dystrophy(DMD) and preserved ejection fraction. On top of angiotensin-converting enzyme inhibitor (ACEI) , half of the participants will receive bisoprolol in combination, while the other half will not receive any beta-blocker.

Full description

By the age of 20 years, almost all the patients with Duchenne muscular dystrophy(DMD) have experienced dilated cardiomyopathy (DCM), a condition that contributes significantly to their morbidity and mortality. Studies have shown ACEI to be an effective therapy for DMD boys with early cardiomyopathy. Although bisoprolol has been demonstrated as an effective neurohumoral drug for adult patients with DCM, few data exist concerning its safety and efficacy for the patients with DMD. The investigators hypothesize that the treatment with bisoprolol in addition to background ACEI therapy for 12 months in participants with DMD and early myocardial damage evident with late gadolinium enhancement and preserved ejection fraction, would blunt decline in left ventricular systolic performance.

Enrollment: 42 estimated patients

Sex: Male

Ages: 7+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Older than(including) 7 years old
• A definite diagnosis of DMD with muscle pathology confirming the expression of dystrophin lower than 5% and/or confirmed mutations in the DMD gene using a clinical accepted technique that completely defines the mutation.
• Using ACEI or ARB for more than 1 month
• Confirmed myocardial damage in one or more left ventricular segments evident by late gadolinium enhancement and preserved left ventricular systolic function(>45%) by cine cardiac MR in 45 days
• Normal renal function
• Holter and blood pressure shows no contraindication of using bisoprolol

Exclusion criteria

• Having metal implanted in body
• Having claustrophobia
• Allergic to gadolinium
• Complicated with other cardiovascular diseases
• Medical history or Holter show bradyarrhythmia like II/III degree atrioventricular block, sick sinus syndrome etc.
• Systolic blood pressure lower than 90mmHg or rest heart rate lower than 75bpm
• Having COPD or asthma history
• Having other complications: tumor, endocrine diseases
• Having beta blockers therapy
• Planned operation in the future 12 months
• Allergic to bisoprolol

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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42 participants in 2 patient groups

bisoprolol fumarate

Experimental group

Description:

In this arm, the participants will receive different dose of bisoprolol fumarate.

Treatment:

Drug: Bisoprolol Fumarate

Control

No Intervention group

Description:

In the control (no beta blocker) group, the patients not taking beta blocker will receive outpatient clinic or video visit every 8 weeks and provide their cardiac symptoms, heart rate, blood pressure and ECG. After 12 months , the patients will repeat cardiac MR besides heart rate, blood pressure, symptoms, ECG,BNP and echocardiography records.