welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
study id #: NCT02834650
condition: Duchenne Muscular Dystrophy
This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in heart health over time in boys with DMD. Currently, there is a lack of sufficiently well characterized cardiac MRI biomarkers that can serve as endpoints for detecting on-target and/or off-target cardiac effects during clinical drug trials for boys with DMD. Consequently, the first objective is to identify and characterize several cardiac MRI biomarkers for boys with DMD.
Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test, Heart Rate, Pulmonary Function Test, Genetic Testing, Repeat MRI scan
mechanism of action: No pharmaceutical intervention
last updated: May 15, 2019
start date: February 1, 2017
estimated completion: January 2021
phase of development: N/A
size / enrollment: 178
The second objective is to use their well-characterized cardiac MRI biomarkers and define their sensitivity for detecting early cardiac involvement. The final objective is to use these validated cardiac MRI biomarkers to better understand the genotype-phenotype correlation in boys with DMD, which to date remain tenuous. The investigators propose a pilot study to explore cardiac genotype-phenotype correlations in boys with DMD and outlier phenotypes using approaches they have pioneered for skeletal muscle.
- Myocardial Tissue Characterization [ Time Frame: 6 months ]
Focal and diffuse fibrosis, intra myocardial fat, edema plus water mobility
- Myocardial Functional Characterization [ Time Frame: 6 months ]
Strain imaging and rotational mechanics
- Genomic Analysis [ Time Frame: 4 years ]
Proposing mechanisms of cardiac dysfunction or protective phenotypes using genomic analysis
• Healthy boys or pediatric patients with DMD age 7 to 21
• Able & willing to complete an approximately 75-minute (or less) MRI exam without sedation or mechanical ventilation
• Drug regimen (if applicable) stable for at least 3 months prior to participation
• Renal insufficiency (GFR<40 mL/min/m2)
• Non-MRI compatible implants (e.g. neurostimulator, pacemaker, implanted cardioverter defibrillator)
• Claustrophobia that prevents an MRI exam
• Known allergy to MRI contrast agents
• Serum potassium level of >5.0 mmol/L
• Signs and symptoms of heart failure
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocortico...The purpose of the study is to assess th...
Catabasis Pharmaceuticals Announces Publication Of Phase 1 Clinical Results Of Edasalonexent (CAT-1004) In Duchenne ...Catabasis Pharmaceuticals, Inc., a clini...
Open Label Extension Study of HT-100 in Patients With DMDThis study is designed to provide 6-mont...
Electrical Impedance Myography in Duchenne Muscular Dystrophy: A Longitudinal StudyObjective:To evaluate the capability of ...
Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2IThe purpose of this study is to investig...
Solid Biosciences Reports Third Quarter 2018 Financial Results And Provides Business UpdateSolid Biosciences Inc. today reported fi...
Finding the Optimum Regimen for Duchenne Muscular DystrophyThe Finding the Optimum Regimen for Duch...
Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne Muscular Dystrop...Catabasis Pharmaceuticals, Inc., a clini...
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...