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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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy (DMD)
Interventions Drug: idebenone
Drug: placebo
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Idebenone 450 mg/ Day Placebo
Hide Arm/Group Description idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals Placebo tablet: One tablet 3 times a day (Tid) with meals
Period Title: Overall Study
Started 13 8
Completed 13 8
Not Completed 0 0
Arm/Group Title Idebenone 450 mg/ Day Placebo Total
Hide Arm/Group Description idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals Placebo tablet: One tablet 3 times a day (Tid) with meals Total of all reporting groups
Overall Number of Baseline Participants 13 8 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants 8 participants 21 participants
<=18 years
13
 100.0%
8
 100.0%
21
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 13 participants 8 participants 21 participants
13.4  (2.1) 10.8  (1.9) 12.4  (2.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants 8 participants 21 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
13
 100.0%
8
 100.0%
21
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Belgium Number Analyzed 13 participants 8 participants 21 participants
13 8 21
1.Primary Outcome
Title 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).
Hide Description
  • 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.

Time Frame baseline and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
At Week 52, data from only 18 patients were available for the primary endpoint analysis due to missing data from 2 patients and the inability to acquire data from a 3rd patient. The efficacy comparison for the primary endpoint was conducted using the LOCF method in the ITT population. In addition, the analysis was repeated using the OC dataset.
Arm/Group Title Idebenone 450 mg/ Day Placebo
Hide Arm/Group Description:
idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals
Placebo tablet: One tablet 3 times a day (Tid) with meals
Overall Number of Participants Analyzed 11 7
Mean (Standard Deviation)
Unit of Measure: % change in peak systolic
104.4  (92.0) 28.9  (40.7)
2.Secondary Outcome
Title Respiratory Function: Forced Vital Capacity (FVC), Forced Expiratory Volume in 1 Second (FEV1), Maximal Inspiratory Pressure (MIP) and Peak Flow (PF)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
3.Secondary Outcome
Title 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)
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
4.Secondary Outcome
Title Safety and Tolerability, Assessed by Adverse Events, Blood and Urine Laboratory Measures, ECG.
Hide Description [Not Specified]
Time Frame 1 year
Outcome Measure Data Not Reported
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Idebenone 450 mg/ Day Placebo
Hide Arm/Group Description idebenone 150 mg tablet: One tablet 3 times a day (Tid) with meals Placebo tablet: One tablet 3 times a day (Tid) with meals
All-Cause Mortality
Idebenone 450 mg/ Day Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Idebenone 450 mg/ Day Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/13 (7.69%)      1/8 (12.50%)    
Injury, poisoning and procedural complications     
Ankle fracture *  1/13 (7.69%)  1 0/8 (0.00%)  0
Femur fracture *  0/13 (0.00%)  0 1/8 (12.50%)  1
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Idebenone 450 mg/ Day Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   12/13 (92.31%)      8/8 (100.00%)    
Gastrointestinal disorders     
Gastrointestinal disorders *  4/13 (30.77%)  5 5/8 (62.50%)  6
General disorders     
General disorders *  2/13 (15.38%)  4 1/8 (12.50%)  1
Infections and infestations     
Infections *  8/13 (61.54%)  19 5/8 (62.50%)  12
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Thomas Meier
Organization: Santhera
Phone: +4161 906 8964
EMail: thomas.meier@santhera.com
Layout table for additonal information
ClinicalTrials.gov Identifier: NCT00654784     History of Changes
Other Study ID Numbers: SNT-II-001
First Submitted: April 3, 2008
First Posted: April 9, 2008
Results First Submitted: June 7, 2011
Results First Posted: July 29, 2011
Last Update Posted: August 1, 2011