Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

Phase 2

Completed

• Conditions: Duchenne Muscular Dystrophy (DMD)
• Interventions: Drug: placebo; Drug: idebenone

• Registration Number: NCT00654784
• Lead Sponsor: Santhera Pharmaceuticals

Brief Summary

Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level. It can protect mitochondria from oxidative damage and boost their impaired function. It is thought that this mechanism will slow decline in heart function that is part of the disease process of Duchenne Muscular Dystrophy (DMD). It is possible that patients may benefit in terms of muscle strength and respiratory function. This pilot trial is designed to investigate this.

Detailed Description

Not available

Study Timeline

• Study Start: 2005-10
• Primary Completion: 2007-08
• Study Completion: 2007-08
• Study First Submitted: 2008-04-03
• Study First Submitted QC: 2008-04-03
• Study First Posted: 2008-04-09
• Results First Submitted: 2011-06-07
• Results First Submitted QC: 2011-06-29
• Status Verified: 2011-07
• Results First Posted: 2011-07-29
• Last Update Submitted: 2011-07-29
• Last Update Posted: 2011-08-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 21

Inclusion Criteria

• Patients 8 - 16 years of age at time of enrolment
• Male
• Presence of cardiac involvement/dysfunction, defined by abnormal peak systolic strain in left ventricle (LV) inferolateral wall
• Confirmed diagnosis of DMD (out of frame dystrophin gene deletion OR absent/<5% dystrophin protein on muscle biopsy; clinical picture consistent of typical DMD)
• If on chronic glucocorticosteroids treatment (deflazacort, prednisone) for DMD (or any other disease) (i.e. concomitant medication): dosage must be stable (unchanged) 6 months prior to inclusion
• If on chronic medication for DMD associated cardiomyopathy (β-blocker, diuretics): dosage must be stable (unchanged) 3 months prior to inclusion
• Ability to provide reproducible repeat quantitative muscle testing (QMT) upper limb score within 15% of first assessment score (at Visit1/Day 1 versus Screening Visit

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Exclusion Criteria

• Symptomatic cardiomyopathy or heart failure
• Asymptomatic but severe cardiac dysfunction on baseline (Screening) evaluation: Fractional shortening (FS) < 20% and/or Ejection fraction (EF) < 40%
• Use of ACE-inhibitors
• Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at Screening
• Previous (6 months or less) participation in any other therapeutic trial for DMD
• Use of coenzymeQ10, idebenone, creatine, glutamine, oxatomide, or any herbal medicines within the last 6 months
• History of significant concomitant illness or significant impairment of renal or hepatic function
• Known individual hypersensitivity to idebenone

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
2	placebo	-
1	idebenone	-

Primary Outcome Measures

Name	Time	Method
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).	baseline and Week 52	* 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.

Secondary Outcome Measures

Name	Time	Method
Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF)	1 year
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)	1 year
Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG.	1 year

Trial Locations

Locations (1)

Children's Hospital, University Hospital; 🇧🇪 Leuven, Belgium