Duchenne Muscular DystrophyPhase 2/3NCT03779646
Bisoprolol in DMD Early Cardiomyopathy
A Phase 2/3 study testing Bisoprolol Fumarate in people with Duchenne Muscular Dystrophy and Cardiomyopathy, Dilated, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Cardiomyopathy, Dilated
- What is being tested?
- Bisoprolol Fumarate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteChina
- Study dates
- 16 Jan 2019to 1 Jul 2024
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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 locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality
Xiaoxiao Guo, Dr.
About this study
Official title: Bisoprolol for Early Cardiomyopathy in Duchenne Muscular Dystrophy: a Randomized, Controlled Trial
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.
Read the 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.
- Being tested
- Bisoprolol Fumarate
- Keywords
- Duchenne muscular dystrophy, Early cardiomyopathy, bisoprolol
- Medical subject headings
- Muscular Dystrophy, Duchenne, Cardiomyopathy, Dilated, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bisoprolol FumarateDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| bisoprolol fumarate | Experimental | In this arm, the participants will receive different dose of bisoprolol fumarate. Receives: Drug: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: baseline and 12 months
Secondary outcomes (6)
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
Measured: baseline and 12 months
Calculate the change of ventricle late gadolinium enhancement area in cardia MR
Calculate the change of ventricle late gadolinium enhancement area in cardia MR from baseline to 12months for each patients
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: baseline and 6months, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2018
- Study start16 Jan 2019
- Primary completion1 Jul 2023 (estimated)
- Study completion1 Jul 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Natural Science Foundation of China, Chinese Academy of Medical Sciences
- Responsible party
- Principal investigator, Xiaoxiao Guo, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03779646
- Sponsor's protocol ID
- PekingUMCH-DMD
- Status last verified
- Sep 2022
- First submitted
- 14 Dec 2018
- Last updated
- 28 Sep 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bisoprolol for Early Cardiomyopathy in Duchenne Muscular Dystrophy: a Randomized, Controlled Trial
- ClinicalTrials.gov ID
- NCT03779646
- Acronym / sponsor ID
- PekingUMCH-DMD
- Keywords
- Duchenne muscular dystrophy, Early cardiomyopathy, bisoprolol
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Collaborators
- National Natural Science Foundation of China, Chinese Academy of Medical Sciences
- Study start
- 16 Jan 2019 (actual)
- Primary completion
- 1 Jul 2023 (estimated)
- Study completion
- 1 Jul 2024 (estimated)
- First posted
- 19 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| bisoprolol fumarate | Experimental | Drug: Bisoprolol FumarateIn 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. |
Who may qualify
- Ages
- 7 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bisoprolol FumarateBisoprolol 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. | Drug | bisoprolol fumarate |
Primary outcome
Calculate the change of left ventricle global longitudinal strain in cardiac MR
Time frame: baseline and 12 months
Calculate the change of left ventricle global longitudinal strain in cardiac MR from baseline to 12months for each patients
6 secondary outcomes
Calculate the change of left ventricular ejection fraction in cardia MR
Time frame: baseline and 12 months
Calculate the change of ventricle late gadolinium enhancement area in cardia MR
Time frame: baseline and 12 months
Calculate the change of the level of high-sensitivity cardiac troponin I
Time frame: baseline and 6months, 12 months
Calculate the change of the level of NT-proBNP
Time frame: baseline and 6months, 12 months
Calculate the change of E/A ratio assessed by echocardiography
Time frame: baseline and 12 months
Change of the resting heart rate
Time frame: baseline and 6months, 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Bisoprolol Fumarate in people with Duchenne Muscular Dystrophy and Cardiomyopathy, Dilated, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Guo, Doctor (+86-010-69155068, xiaoxiaoguopumch@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Calculate the change of left ventricle global longitudinal strain in cardiac MR (time frame: baseline and 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2022.