welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy – ACE Inhibitor Therapy Trial
study id #: NCT02432885
condition: Myocardial Fibrosis, Muscular Dystrophies
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.
mechanism of action: ACE inhibitor and beta-blocker to prevent cardiac function deterioration
last updated: November 22, 2018
start date: June 2009
estimated completion: June 2012
phase of development: Phase 3
size / enrollment: 76
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.
- Quantitative Myocardial Fibrosis by CMR in patients with and without ACE inhibitor therapy [ Time Frame: 2 years ]
Progression of myocardial fibrosis
- Specific genetic mutations as predictors of cardiac involvement [ Time Frame: 2 years ]
Relation of dystrophin gene site mutations in exons <45 relation and the extent of myocardial fibrosis measured by cardiac magnetic resonance
inclusion criteria: Patients with biopsy-proven Muscular Dystrophy of Duchenne or Becker
exclusion criteria: Contraindications to cardiovascular magnetic resonance imaging
Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyThis study is a long-term study of atalu...
Design of a phase 3 trial to evaluate the long-term efficacy and safety of ataluren in patients with nonsense mutati...Ataluren is conditionally approved by th...
Study of ataluren in previously treated participants with nonsense mutation dystrophinopathy (nmDBMD)The objective of this study is to assess...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsHappy New Year! As 2019 kicks off, our P...
Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical TrialObjective: To assess the effect of earl...
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double...Background: Duchenne muscular dystrophy...
Catabasis Quarterly: Updates on edasalonexent and clinical trialsWe are pleased to announce that we have ...