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

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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

Tracking Information
First Submitted Date  ICMJE April 3, 2008
First Posted Date  ICMJE April 9, 2008
Results First Submitted Date  ICMJE June 7, 2011
Results First Posted Date  ICMJE July 29, 2011
Last Update Posted Date August 1, 2011
Study Start Date  ICMJE October 2005
Actual Primary Completion Date August 2007   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 29, 2011)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: April 3, 2008)
The relative change from Baseline (at Screening) to Week 52 in peak systolic radial strain of the LV inferolateral wall, assessed by Color Doppler Myocardial Imaging (CDMI). [ Time Frame: 1 year ]
Change History Complete list of historical versions of study NCT00654784 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 3, 2008)
  • 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 ]
  • 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 ]
  • Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG. [ Time Frame: 1 year ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Official Title  ICMJE 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
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy (DMD)
Intervention  ICMJE
  • Drug: idebenone
    idebenone 450 mg/day (150 mg three times a day)
  • Drug: placebo
Study Arms  ICMJE
  • Experimental: 1
    Intervention: Drug: idebenone
  • Placebo Comparator: 2
    Intervention: Drug: placebo
Publications * Buyse GM, Goemans N, van den Hauwe M, Thijs D, de Groot IJ, Schara U, Ceulemans B, Meier T, Mertens L. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscul Disord. 2011 Jun;21(6):396-405. doi: 10.1016/j.nmd.2011.02.016. Epub 2011 Mar 23.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 3, 2008)
21
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE August 2007
Actual Primary Completion Date August 2007   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 8 Years to 16 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00654784
Other Study ID Numbers  ICMJE SNT-II-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE Santhera Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Gunnar Buyse, MD PhD Universitaire Ziekenhuizen Leuven
PRS Account Santhera Pharmaceuticals
Verification Date July 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP