Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy (DELPHI)
Duchenne Muscular Dystrophy (DMD)
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy|
- The Relative Change in Peak Systolic Radial Strain of the Left Ventricle (LV) Inferolateral Wall From Baseline (at Screening) to Week 52, Assessed by Color Doppler Myocardial Imaging (CDMI). [ Time Frame: baseline and Week 52 ] [ Designated as safety issue: No ]
- Assessing the peak systolic radial strain of the left ventricle inferolateral wall is used to characterize the cardiac involvement in the DMD patients.
- Color Doppler Myocardial Imaging technique is used to quantify regional myocardial function.
The cardiac involvement in DMD is characterized by degeneration, atrophy and fibrosis of the myocardium, leading to dilated cardiomyopathy. The process begins in the posterolateral wall of the left ventricle, with septal involvement appearing at later stages.
- Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Skeletal Muscle Strength (Upper Limb, Right and Left): Hand Grip, Elbow Flexors and Elbow Extensors (Upper Limb Score) Timed Walking Test (10 Metres) (Ambulant Patients Only) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG. [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
|Study Start Date:||October 2005|
|Study Completion Date:||August 2007|
|Primary Completion Date:||August 2007 (Final data collection date for primary outcome measure)|
idebenone 450 mg/day (150 mg three times a day)
|Placebo Comparator: 2||Drug: placebo|
Please refer to this study by its ClinicalTrials.gov identifier: NCT00654784
|Children's Hospital, University Hospital|
|Principal Investigator:||Gunnar Buyse, MD PhD||Universitaire Ziekenhuizen Leuven|