Myocardial FibrosisPhase 3NCT02432885
Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial
A Phase 3 study testing Enalapril in people with Myocardial Fibrosis and Muscular Dystrophies, run by InCor Heart Institute.
Trial summary
This trial intends to evaluate myocardial Fibrosis progression in Duchenne and Becker Muscular Dystrophy, as well the influence of ACE inhibitors in fibrosis progression. Additionally, this study aims to determine genetic predictors of cardiac involvement in these dystrophies. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Myocardial Fibrosis, Muscular Dystrophies
- What is being tested?
- Enalapril
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- No sites listed yet
- Study dates
- Jun 2009to Jun 2013
- Phase
- Phase 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker
Who cannot join
- Contraindications to cardiovascular magnetic resonance imaging
About this study
Official title: Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy
This trial intends to evaluate myocardial Fibrosis progression in Duchenne and Becker Muscular Dystrophy, as well the influence of ACE inhibitors in fibrosis progression. Additionally, this study aims to determine genetic predictors of cardiac involvement in these dystrophies.
Read the detailed description
Duchenne and Becker muscular dystrophies (DMD/BMD) are diseases characterized by progressive skeletal muscle degeneration and replacement by fibrofatty tissue. Data on cardiac involvement (defined as myocardial fibrosis), effect of ACE-inhibitors and specific genetic mutations on myocardial involvement detected by cardiac magnetic resonance (CMR) is lacking.
The study will include 76 patients with DMD/BMD. All patients will be referred to two CMRs for assessment of ventricular function and myocardial fibrosis. Patients with myocardial fibrosis and normal left ventricle ejection fraction (LVEF) will be randomized into two groups, each group receiving ACE-inhibitor treatment or no treatment for cardiomyopathy. A genetic profile will be performed in every patient to identify possible mutations related to cardiac involvement.
- Conditions
- Myocardial Fibrosis, Muscular Dystrophies
- Being tested
- Enalapril
- Keywords
- cardiac magnetic resonance, myocardial fibrosis, muscular dystrophies
- Medical subject headings
- Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EnalaprilDrug
up to 20mg bid
| Study group | Role | What they receive |
|---|---|---|
| ACE inhibitor | Experimental | ACE inhibitor (enalapril up to 20mg BID), in patients with preserved EF (LVEF grater than 50%) and with detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance, randomized to therapy or not. Receives: Drug: Enalapril |
| Control | No intervention | Patients with preserved EF (LVEF grater than 50%) and with no detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
Progression of myocardial fibrosis
Measured: 2 years
Secondary outcomes (1)
Specific genetic mutations as predictors of cardiac involvement
Relation of dystrophin gene site mutations in exons <45 relation and the extent of myocardial fibrosis measured by cardiac magnetic resonance
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2015 (estimated)
- Study startJun 2009
- Primary completionJun 2012
- Study completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myocardial FibrosisStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- InCor Heart Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Federal University of Minas Gerais, University of Sao Paulo
- Responsible party
- Principal investigator, Carlos Eduardo Rochitte, InCor Heart Institute
- Investigators
- Carlos E Rochitte, MD, PhD, Principal investigator, InCor, Heart Institute, University of Sao Paulo Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02432885
- Sponsor's protocol ID
- 1095/08
- Status last verified
- Apr 2015
- First submitted
- 24 Mar 2015
- Last updated
- 4 May 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Silva MC, Magalhaes TA, Meira ZM, Rassi CH, Andrade AC, Gutierrez PS, Azevedo CF, Gurgel-Giannetti J, Vainzof M, Zatz M, Kalil-Filho R, Rochitte CE. Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial. JAMA Cardiol. 2017 Feb 1;2(2):190-199. doi: 10.1001/jamacardio.2016.4801.
PubMed 27926769
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy
- ClinicalTrials.gov ID
- NCT02432885
- Acronym / sponsor ID
- 1095/08
- Conditions
- Myocardial Fibrosis, Muscular Dystrophies
- Keywords
- cardiac magnetic resonance, myocardial fibrosis, muscular dystrophies
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- InCor Heart InstituteUniversity, hospital or other
- Collaborators
- Federal University of Minas Gerais, University of Sao Paulo
- Study start
- Jun 2009
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 4 May 2015 (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.
This trial intends to evaluate myocardial Fibrosis progression in Duchenne and Becker Muscular Dystrophy, as well the influence of ACE inhibitors in fibrosis progression. Additionally, this study aims to determine genetic predictors of cardiac involvement in these dystrophies.
Detailed description
Duchenne and Becker muscular dystrophies (DMD/BMD) are diseases characterized by progressive skeletal muscle degeneration and replacement by fibrofatty tissue. Data on cardiac involvement (defined as myocardial fibrosis), effect of ACE-inhibitors and specific genetic mutations on myocardial involvement detected by cardiac magnetic resonance (CMR) is lacking.
The study will include 76 patients with DMD/BMD. All patients will be referred to two CMRs for assessment of ventricular function and myocardial fibrosis. Patients with myocardial fibrosis and normal left ventricle ejection fraction (LVEF) will be randomized into two groups, each group receiving ACE-inhibitor treatment or no treatment for cardiomyopathy. A genetic profile will be performed in every patient to identify possible mutations related to cardiac involvement.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ACE inhibitor | Experimental | Drug: EnalaprilACE inhibitor (enalapril up to 20mg BID), in patients with preserved EF (LVEF grater than 50%) and with detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance, randomized to therapy or not. |
| Control | No intervention | –Patients with preserved EF (LVEF grater than 50%) and with no detectable delayed enhancement (myocardial fibrosis) in cardiac magnetic resonance |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker
Key exclusion criteria (1 listed)
- Contraindications to cardiovascular magnetic resonance imaging
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 |
|---|---|---|
| Enalaprilup to 20mg bid | Drug | ACE inhibitor |
Primary outcome
Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy
Time frame: 2 years
Progression of myocardial fibrosis
1 secondary outcome
Specific genetic mutations as predictors of cardiac involvement
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 May 2015.
- Derived from this study: PubMed 27926769Silva MC, Magalhaes TA, Meira ZM, Rassi CH, Andrade AC, Gutierrez PS, Azevedo CF, Gurgel-Giannetti J, Vainzof M, Zatz M, Kalil-Filho R, Rochitte CE. Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial. JAMA Cardiol. 2017 Feb 1;2(2):190-199. doi: 10.1001/jamacardio.2016.4801.
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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.
Carlos E Rochitte, MD, PhD principal investigator
InCor, Heart Institute, University of Sao Paulo Medical School
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Enalapril in people with Myocardial Fibrosis and Muscular Dystrophies, run by InCor Heart Institute.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 4 May 2015. Status can change between updates, so confirm with the study contact before making plans.
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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02432885, which also lists 1 linked publication.
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 4 May 2015.