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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00654784
Recruitment Status : Completed
First Posted : April 9, 2008
Results First Posted : July 29, 2011
Last Update Posted : August 1, 2011
Sponsor:
Information provided by:
Santhera Pharmaceuticals

Study Description
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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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy (DMD) Drug: idebenone Drug: placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : October 2005
Actual Primary Completion Date : August 2007
Actual Study Completion Date : August 2007

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 1 Drug: idebenone
idebenone 450 mg/day (150 mg three times a day)
Placebo Comparator: 2 Drug: placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
    • 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 :
  1. 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 ]
  2. 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 ]
  3. Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG. [ Time Frame: 1 year ]

Eligibility Criteria
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Ages Eligible for Study:   8 Years to 16 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00654784


Locations
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Belgium
Children's Hospital, University Hospital
Leuven, Belgium
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
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Principal Investigator: Gunnar Buyse, MD PhD Universitaire Ziekenhuizen KU Leuven
More Information
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Publications of Results:

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ClinicalTrials.gov Identifier: NCT00654784    
Other Study ID Numbers: SNT-II-001
First Posted: April 9, 2008    Key Record Dates
Results First Posted: July 29, 2011
Last Update Posted: August 1, 2011
Last Verified: July 2011
Keywords provided by Santhera Pharmaceuticals:
Duchenne Muscular Dystrophy
DMD
Duchenne
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
 Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs