Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| National Natural Science Foundation of China | OTHER_GOV |
| Chinese Academy of Medical Sciences | OTHER |
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| bisoprolol fumarate | Experimental | In this arm, the participants will receive different dose of bisoprolol fumarate. |
|
| Control | No Intervention | 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. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Bisoprolol Fumarate | Drug | Bisoprolol was initiated at a dose of 1.25 mg every 24hr. At subsequent biweekly visits, the bisoprolol dose was increased 1.25mg progressively until a daily dose of 0.2mg/kg or the maximum tolerated dose (The rest heart rate <75bpm and systolic blood pressure <90mmHg) is achieved. If the participant feel dizziness or the rest heart rate below 60 bpm or systolic blood pressure below 85mmHg or there is any new onset contraindication to bisoprolol, the doctor will decide to return back to the previous dose or stop bisoprolol. Each time the dose is increased, the medication is administered in an outpatient clinic setting, with assessment of the participant's heart rate, blood pressure, symptoms and ECG. Once reach the target dose, the patients will be followed up every 8 weeks in an face-to-face or video visit. After 12 months , the participants will repeat cardiac MR besides heart rate, blood pressure, symptoms, ECG and brain natriuretic peptide(BNP), echocardiography records. |
| Measure | Description | Time Frame |
|---|---|---|
| Calculate the change of left ventricle global longitudinal strain in cardiac MR | Calculate the change of left ventricle global longitudinal strain in cardiac MR from baseline to 12months for each patients | baseline and 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Calculate the change of left ventricular ejection fraction in cardia MR | Calculate the change of left ventricular ejection fraction in cardia MR from baseline to 12months for each patients | baseline and 12 months |
| Calculate the change of ventricle late gadolinium enhancement area in cardia MR |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with All cause mortality, cardiac death, or hospitalized due to heart problem | Number of Subjects with All cause mortality, cardiac death, or hospitalized due to heart problem at the end of study | 12 months |
| Number of participants with bisoprolol-related adverse events as assessed by the following definition |
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Guo, Doctor | Contact | +86-010-69155068 | xiaoxiaoguopumch@163.com |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Recruiting | Beijing | Beijing Municipality | China |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D002311 | Cardiomyopathy, Dilated |
| D009202 | Cardiomyopathies |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D017298 | Bisoprolol |
| ID | Term |
|---|---|
| D050198 | Phenoxypropanolamines |
| D011412 | Propanolamines |
| D000605 | Amino Alcohols |
| D000438 | Alcohols |
Not provided
Not provided
This is a randomized, controlled trial.Enrolled participants should receive a kind of ACEI drug for more than 1 month and the dose should be fixed. Then the participants were randomly assigned (1:1) to receive either bisoprolol or not any beta blocker.
Not provided
Not provided
The investigator, care providers and participants will know whether the participants use the trial drug（bisoprolol） or not. But the outcome assessor who will analysis the results of the cardiac MR and echocardiography images from the participants will be blinded to the grouping situation.
|
Calculate the change of ventricle late gadolinium enhancement area in cardia MR from baseline to 12months for each patients |
| baseline and 12 months |
| Calculate the change of the level of high-sensitivity cardiac troponin I | Calculate the change of the level of high-sensitivity cardiac troponin I from baseline to 6months and 12months for each patients | baseline and 6months, 12 months |
| Calculate the change of the level of NT-proBNP | Calculate the change of the level of NT-proBNP from baseline to 6months and 12months for each patients | baseline and 6months, 12 months |
| Calculate the change of E/A ratio assessed by echocardiography | Calculate the change of diastolic dysfunction (E/A ratio) assessed by echocardiography from baseline to 12months for each patients | baseline and 12 months |
| Change of the resting heart rate | Calculate the change of the resting heart rate from baseline to 6months and 12months for each patients | baseline and 6months, 12 months |
Bisoprolol treatment-related adverse events including the dizziness, bradyarrhythmia (resting heart rate lower than 55bpm)，II and III degree atrioventricular block，hypotension |
| 12 months |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006332 | Cardiomegaly |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D000083083 | Laminopathies |
| D009930 |
| Organic Chemicals |
| D020005 | Propanols |
| D000588 | Amines |