Duchenne Muscular DystrophyPhase 4NCT00819845
Ramipril Versus Carvedilol in Duchenne and Becker Patients
A Phase 4 study testing carvedilol and ramipril in people with Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by Catholic University, Italy at 1 site in Italy.
Trial summary
Data on preventive therapy in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) affected individuals without cardiac involvement are very limited and currently lacking regard both ACE-inhibitors and Beta-Blockers in Becker Muscular Dystrophy and for the latter even in Duchenne Muscular Dystrophy… Read more about this study
- Who is it for?
- Ages 2 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- What is being tested?
- carvedilol and ramipril
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 194 participants
- Where
- 1 siteItaly
- Study dates
- Dec 2008to Dec 2016
- Phase
- Phase 4
- 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 2 to 45 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Immunohystochemical and molecular diagnosis of Duchenne and Becker muscular dystrophy.
- Not evidence of clinical cardiomyopathy,normal 2D-echocardiography with normal systolic,WMSI = 1) and diastolic function.
- DMD patients treated with steroid therapy.
- All DMD and BMD patients are not treated with cardiological therapy (ACE-inhibitors, ARBs or Beta-Blockers).
- Written informed consent to study participation (with serial visit, CMR and echocardiographic study) is required from all patients themselves, as well as their parent or guardian and healthy-control subjects.
Who cannot join
- Failure to obtain informed consent from patients, parents or guardians.
- Any controindications to carvedilol or ramipril treatment (bronchial asthma, diabetes, any degree of renal failure (all patients are required to have a normal creatinine level and clearance).
- in BMD patients ECG changes suggestive of ischemic heart disease, left bundle-branch block, atrial flutter/fibrillation, ventricular arrhythmias, any degree of atrioventricular block and left ventricular (LV) hypertrophy. Aspecific ST changes will be not considered as electrocardiographic exclusion criteria both in DMD and BMD patients.
- In BMD patients exclusion criteria will be also hypertension and valvular heart disease other than trivial.
- DMD and BMD patients requiring ventilatory (non-invasive or invasive) assistance.
- Presence of systolic and/or diastolic dysfunction detected by 2D-Echocardiography.
Show all 10Show fewer
- Presence of any contraindications to CMR (including any history of claustrophobia).
- Patients under the age of 2 years.
- Renal failure, even mild.
- Patient unable or unwilling to attend the follow-up and tests, in the opinion of local study principal investigator, (children not willing to perform CMR will not be enrolled).
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
Unione Italiana lotta Distrofia Muscolare
Rome, 00167
Vincenzo Giglio, MD, PhD, 39-6-6604881, giglio.echo@libero.it
Fortunato Mangiola, MD, 39-6-6604881, fortunato.mangiola@uildmlazio.org
About this study
Official title: Effects of Cardioprotective Therapy, Carvedilol vs Ramipril, in Patients Affected by Duchenne and Becker Muscular Dystrophy. Clinical Significance and Prognostic Value of Cardiac Magnetic Resonance Study.
Data on preventive therapy in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) affected individuals without cardiac involvement are very limited and currently lacking regard both ACE-inhibitors and Beta-Blockers in Becker Muscular Dystrophy and for the latter even in Duchenne Muscular Dystrophy patients. Thus, the study aim is to compare the efficacy of carvedilol vs ramipril on myocardial tissue properties and heart function, performing CMR and myocardial Ultrasound Tissue Characterisation analysis.
Read the detailed description
This protocol represent an open randomized and prospective trial, designed to answer the specific question regarding the role of the cardioprotective therapy in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy patients. In this light, CMR could provide relevant data, reinforcing the scientific background, to start early (particularly in BMD patients in whom this is still a debated question) a cardioprotective treatment with carvedilol or ramipril.Finally,this clinical trial will clarify whether a preventive therapy may be helpful on the clinical outcome, both in reducing myocardial fibrosis and preventing the progression towards the cardiomyopathy.
- Being tested
- carvedilol, ramipril
- Keywords
- Duchenne, Becker muscular dystrophy, cardioprotective therapy, cardiac magnetic resonance, ultrasound tissue characterization
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
carvedilolDrug
Also called Beta-Blocker
carvedilol vs ramipril
ramiprilDrug
Also called ACE-inhibitor
carvedilol vs ramipril
| Study group | Role | What they receive |
|---|---|---|
| Ramipril | Experimental | Not described Receives: Drug: ramipril |
| Carvedilol | Experimental | Not described Receives: Drug: carvedilol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Left ventricular Ejection Fraction, systolic and diastolic left ventricular volumes and LGE (as a quantitative measure) detected by MRI and myocardial Ultrasound Tissue Characterisation data by Echocardiography.
Measured: 1 year
Secondary outcomes (1)
Prevalence of LGE in DMD and BMD patients,the effects of pharmacological therapy both on LGE evolution and myocardial UTC analysis.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2009 (estimated)
- Study startDec 2008
- Primary completionJun 2009
- Study completionDec 2016 (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.
- 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 4
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 194 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Catholic University, Italy
- Sponsor type
- University, hospital or other
- Investigators
- Vincenzo Giglio, MD, PhD, Principal investigator, Uildm, Rome
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00819845
- Sponsor's protocol ID
- Uildm Rome
- Status last verified
- Jan 2009
- First submitted
- 8 Jan 2009
- Last updated
- 28 Jan 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Cardioprotective Therapy, Carvedilol vs Ramipril, in Patients Affected by Duchenne and Becker Muscular Dystrophy. Clinical Significance and Prognostic Value of Cardiac Magnetic Resonance Study.
- ClinicalTrials.gov ID
- NCT00819845
- Acronym / sponsor ID
- Uildm Rome
- Keywords
- Duchenne, Becker muscular dystrophy, cardioprotective therapy, cardiac magnetic resonance, ultrasound tissue characterization
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 194 participants (estimated)
- Lead sponsor
- Catholic University, ItalyUniversity, hospital or other
- Study start
- Dec 2008
- Primary completion
- Jun 2009 (actual)
- Study completion
- Dec 2016 (estimated)
- First posted
- 9 Jan 2009 (estimated)
- 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.
Data on preventive therapy in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) affected individuals without cardiac involvement are very limited and currently lacking regard both ACE-inhibitors and Beta-Blockers in Becker Muscular Dystrophy and for the latter even in Duchenne Muscular Dystrophy patients. Thus, the study aim is to compare the efficacy of carvedilol vs ramipril on myocardial tissue properties and heart function, performing CMR and myocardial Ultrasound Tissue Characterisation analysis.
Detailed description
This protocol represent an open randomized and prospective trial, designed to answer the specific question regarding the role of the cardioprotective therapy in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy patients. In this light, CMR could provide relevant data, reinforcing the scientific background, to start early (particularly in BMD patients in whom this is still a debated question) a cardioprotective treatment with carvedilol or ramipril.Finally,this clinical trial will clarify whether a preventive therapy may be helpful on the clinical outcome, both in reducing myocardial fibrosis and preventing the progression towards the cardiomyopathy.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ramipril | Experimental | Drug: ramipril |
| Carvedilol | Experimental | Drug: carvedilol |
Who may qualify
- Ages
- 2 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Immunohystochemical and molecular diagnosis of Duchenne and Becker muscular dystrophy.
- Not evidence of clinical cardiomyopathy,normal 2D-echocardiography with normal systolic,WMSI = 1) and diastolic function.
- DMD patients treated with steroid therapy.
- All DMD and BMD patients are not treated with cardiological therapy (ACE-inhibitors, ARBs or Beta-Blockers).
- Written informed consent to study participation (with serial visit, CMR and echocardiographic study) is required from all patients themselves, as well as their parent or guardian and healthy-control subjects.
Key exclusion criteria (10 listed)
- Failure to obtain informed consent from patients, parents or guardians.
- Any controindications to carvedilol or ramipril treatment (bronchial asthma, diabetes, any degree of renal failure (all patients are required to have a normal creatinine level and clearance).
- in BMD patients ECG changes suggestive of ischemic heart disease, left bundle-branch block, atrial flutter/fibrillation, ventricular arrhythmias, any degree of atrioventricular block and left ventricular (LV) hypertrophy. Aspecific ST changes will be not considered as electrocardiographic exclusion criteria both in DMD and BMD patients.
- In BMD patients exclusion criteria will be also hypertension and valvular heart disease other than trivial.
- DMD and BMD patients requiring ventilatory (non-invasive or invasive) assistance.
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 |
|---|---|---|
| carvedilolAlso: Beta-Blockercarvedilol vs ramipril | Drug | Carvedilol |
| ramiprilAlso: ACE-inhibitorcarvedilol vs ramipril | Drug | Ramipril |
Primary outcome
Left ventricular Ejection Fraction, systolic and diastolic left ventricular volumes and LGE (as a quantitative measure) detected by MRI and myocardial Ultrasound Tissue Characterisation data by Echocardiography.
Time frame: 1 year
1 secondary outcome
Prevalence of LGE in DMD and BMD patients,the effects of pharmacological therapy both on LGE evolution and myocardial UTC analysis.
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jan 2016.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
Vincenzo Giglio, MD, PhD principal investigator
Uildm, Rome
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing carvedilol and ramipril in people with Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, run by Catholic University, Italy at 1 site in Italy.
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 5 inclusion and 10 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 Jan 2016. 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 Italy. 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 phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 Vincenzo Giglio, MD, PhD (39-6-6604881, giglio.echo@libero.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Left ventricular Ejection Fraction, systolic and diastolic left ventricular volumes and LGE (as a quantitative measure) detected by MRI and myocardial Ultrasound Tissue Characterisation data by Echocardiography. (time frame: 1 year). 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 Jan 2016.