Bisoprolol in DMD Early Cardiomyopathy

Phase 2

Recruiting

• Conditions: Duchenne Muscular Dystrophy; Cardiomyopathy, Dilated
• Interventions: Drug: Bisoprolol Fumarate

• Registration Number: NCT03779646
• Lead Sponsor: Peking Union Medical College Hospital

Brief Summary

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.

Detailed 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.

Study Timeline

• Study First Submitted: 2018-12-14
• Study First Submitted QC: 2018-12-16
• Study First Posted: 2018-12-19
• Study Start: 2019-01-16
• Status Verified: 2022-09
• Last Update Submitted: 2022-09-27
• Last Update Posted: 2022-09-28
• Primary Completion: 2023-07-01
• Study Completion: 2024-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 42

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
bisoprolol fumarate	Bisoprolol Fumarate	In this arm, the participants will receive different dose of bisoprolol fumarate.

Primary Outcome Measures

Name	Time	Method
Calculate the change of left ventricle global longitudinal strain in cardiac MR	baseline and 12 months	Calculate the change of left ventricle global longitudinal strain in cardiac MR from baseline to 12months for each patients

Secondary Outcome Measures

Name	Time	Method
Calculate the change of left ventricular ejection fraction in cardia MR	baseline and 12 months	Calculate the change of left ventricular ejection fraction in cardia MR from baseline to 12months for each patients
Calculate the change of the level of high-sensitivity cardiac troponin I	baseline and 6months, 12 months	Calculate the change of the level of high-sensitivity cardiac troponin I from baseline to 6months and 12months for each patients
Change of the resting heart rate	baseline and 6months, 12 months	Calculate the change of the resting heart rate from baseline to 6months and 12months for each patients
Calculate the change of the level of NT-proBNP	baseline and 6months, 12 months	Calculate the change of the level of NT-proBNP from baseline to 6months and 12months for each patients
Calculate the change of E/A ratio assessed by echocardiography	baseline and 12 months	Calculate the change of diastolic dysfunction (E/A ratio) assessed by echocardiography from baseline to 12months for each patients
Calculate the change of ventricle late gadolinium enhancement area in cardia MR	baseline and 12 months	Calculate the change of ventricle late gadolinium enhancement area in cardia MR from baseline to 12months for each patients

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China