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Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) (DELOS)

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: NCT01027884
Recruitment Status : Completed
First Posted : December 9, 2009
Results First Posted : October 19, 2015
Last Update Posted : October 19, 2015
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Muscular Dystrophy, Duchenne
Ambulatory Care
Interventions Drug: Placebo
Drug: Idebenone
Enrollment 65
Recruitment Details Recruiting centres were in Belgium, Germany, the Netherlands, Switzerland, France, Sweden, Austria, Italy, Spain, and the USA. Patients were enrolled between July 27, 2009 (study start date), and Dec 14, 2012; the study end date (last patient completed the study) was Jan 14, 2014.
Pre-assignment Details 65 patients were randomly assigned and two patients were allocated to the same treatment as their randomly assigned siblings. One patient never took study medication, resulting in 66 patients who were treated and included in the safety population (34 in the placebo group and 32 in the idebenone group).
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description Two matching placebo tablets were taken three times a day with meals Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily).
Period Title: Overall Study
Started 34 32
Completed 30 25
Not Completed 4 7
Reason Not Completed
Adverse Event             2             1
Protocol Violation             1             0
Non-compliance             1             1
Withdrawal by Subject             0             1
Lost to Follow-up             0             1
Spinal fixation surgery             0             3
Arm/Group Title Placebo Idebenone Total
Hide Arm/Group Description Two matching placebo tablets were taken three times a day with meals Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily). Total of all reporting groups
Overall Number of Baseline Participants 34 32 66
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 34 participants 32 participants 66 participants
15  (2.5) 13.5  (2.7) 14.3  (2.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 32 participants 66 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
34
 100.0%
32
 100.0%
66
 100.0%
1.Primary Outcome
Title Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
Hide Description Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 52
Time Frame Baseline and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
This analysis was performed on the ITT population(n=64). This population included all randomized patients who received at least one dose of the study medication and provided at least one post-Baseline assessment. It excluded siblings who had been allocated to the same study treatment as a randomized sibling.
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:
Two matching placebo tablets were taken three times a day with meals
Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily).
Overall Number of Participants Analyzed 33 31
Geometric Mean (95% Confidence Interval)
Unit of Measure: percentage
-8.84
(-12.73 to -4.95)
-2.57
(-6.68 to 1.54)
2.Secondary Outcome
Title Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Hide Description Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52
Time Frame Baseline and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
This analysis was performed on the ITT population(n=64). This population included all randomized patients who received at least one dose of the study medication and provided at least one post-Baseline assessment. It excluded siblings who had been allocated to the same study treatment as a randomized sibling.
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:

Placebo 900 mg/day

Placebo: Placebo (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals

Idebenone 900 mg/day

Idebenone: Idebenone (900 mg/day) 2 tabl (150 mg each) x 3 times orally with meals

Overall Number of Participants Analyzed 33 31
Mean (95% Confidence Interval)
Unit of Measure: percentage of Predicted FVC
-8.95
(-11.47 to -6.42)
-5.67
(-8.36 to -2.99)
3.Secondary Outcome
Title Change From Baseline to Week 52 in Muscle Strength
Hide Description

The change from Baseline to Week 52 in muscle strength as measured by Hand-Held Myometry (HHM) was performed following standardized procedures. As almost all patients were non-ambulatory, only analyses of upper limb muscle strength were performed. Results for elbow flexors and for elbow extensors are reported below.The highest value of 3 consecutive measurements with an interval of at least 10 seconds were recorded.

The HHM was measured using MicroFET2, a digital hand held muscle tester. The selected unit of measure was Newtons (N).

Time Frame Baseline and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
The number of patients (N) in each treatment group is the number of patients with baseline assessments
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:
Two matching placebo tablets were taken three times a day with meals
Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily).
Overall Number of Participants Analyzed 27 25
Mean (95% Confidence Interval)
Unit of Measure: Newtons
Elbow Flexors
0.13
(-4.16 to 4.41)
-2.32
(-6.73 to 2.09)
Elbow Extensors
1.32
(-2.53 to 5.18)
0.26
(-3.95 to 4.46)
4.Secondary Outcome
Title Change From Baseline to Week 52 in Quality of Life Assessed by PedsQL™ Paediatric Quality of Life Inventory
Hide Description

PedsQL Quality of Life Inventory contains paediatric HRQOL measurements: Physical, Emotional,Social and School Functioning.

Item Scaling:

5-point Likert scale from 0 (Never) to 4 (Almost always). 3-point scale: 0 (Not at all), 2 (Sometimes) and 4 (A lot) for the Young Child (ages 5-7).

Scores are transformed on a scale from 0 to 100 ( 0=100, 1=75, 2=50, 3=25, 4=0) Total Score: Sum of all the items over the number of items answered on all the Scales.

The values reported below are overall scores on Paediatric Quality of Life Inventory in Child/Teen Report. These scores were obtained by averaging scores for all the described subscales. The overall scores range between 0-100 with 0 = worst outcome and 100= best outcome

Time Frame Baseline and Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
The number of patients (N) in each treatment group is the number of patients with Baseline assessments.
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:
Two matching placebo tablets were taken three times a day with meals
Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily).
Overall Number of Participants Analyzed 33 30
Mean (95% Confidence Interval)
Unit of Measure: units on a scale
2.46
(-1.77 to 6.69)
-1.34
(-5.99 to 3.31)
5.Secondary Outcome
Title Percentage of Patients Reporting Adverse Events
Hide Description [Not Specified]
Time Frame 52 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description:
Two matching placebo tablets were taken three times a day with meals
Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily).
Overall Number of Participants Analyzed 34 32
Measure Type: Number
Unit of Measure: percentage of patients reporting AEs
94.1 93.8
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Idebenone
Hide Arm/Group Description Two matching placebo tablets were taken three times a day with meals Two150 mg tablets were taken three times a day with meals (total dose 900 mg daily).
All-Cause Mortality
Placebo Idebenone
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Idebenone
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/34 (14.71%)      2/32 (6.25%)    
Gastrointestinal disorders     
Vomitig  1/34 (2.94%)  0/32 (0.00%) 
General disorders     
Pyrexia  1/34 (2.94%)  1 0/32 (0.00%)  0
Infections and infestations     
Pneumonia  2/34 (5.88%)  3 0/32 (0.00%)  0
Nasopharyngitis  1/34 (2.94%)  1 0/32 (0.00%)  0
Injury, poisoning and procedural complications     
Femur fracture  1/34 (2.94%)  1 0/32 (0.00%)  0
Metabolism and nutrition disorders     
Dehydration  1/34 (2.94%)  1 0/32 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Tendinous contracture  1/34 (2.94%)  2 0/32 (0.00%)  0
Psychiatric disorders     
Sleep apnoea syndrome  0/34 (0.00%)  1/32 (3.13%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory failure  1/34 (2.94%)  1 0/32 (0.00%)  0
Pulmunary microemboli  1/34 (2.94%)  1 0/32 (0.00%)  0
Respiratory failure  0/34 (0.00%)  0 1/32 (3.13%)  1
Skin and subcutaneous tissue disorders     
Urticaria  0/34 (0.00%)  0 1/32 (3.13%)  1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Placebo Idebenone
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   32/34 (94.12%)      30/32 (93.75%)    
Blood and lymphatic system disorders     
Blood phosporus increased  3/34 (8.82%)  4 1/32 (3.13%)  1
Cardiac disorders     
Left ventricular failure  1/34 (2.94%)  1 3/32 (9.38%)  3
Electrocardiogram abnormal  1/34 (2.94%)  1 2/32 (6.25%)  3
Gastrointestinal disorders     
Diarrhoea  4/34 (11.76%)  6 8/32 (25.00%)  10
Constipation  6/34 (17.65%)  6 3/32 (9.38%)  4
Abdominal pain  3/34 (8.82%)  5 3/32 (9.38%)  4
Nausea  2/34 (5.88%)  2 1/32 (3.13%)  2
Vomiting  2/34 (5.88%)  3 1/32 (3.13%)  1
General disorders     
Pyrexia  3/34 (8.82%)  4 5/32 (15.63%)  6
Influenza like illness  1/34 (2.94%)  2 2/32 (6.25%)  2
Infections and infestations     
Nasopharyngitis  9/34 (26.47%)  11 8/32 (25.00%)  12
Upper respiratory tract infection  6/34 (17.65%)  10 2/32 (6.25%)  2
Gastroenteritis  1/34 (2.94%)  1 6/32 (18.75%)  7
Rhinitis  6/34 (17.65%)  8 1/32 (3.13%)  1
Otitis media  0/34 (0.00%)  0 3/32 (9.38%)  3
Musculoskeletal and connective tissue disorders     
Back pain  4/34 (11.76%)  6 2/32 (6.25%)  2
Scoliosis  1/34 (2.94%)  1 2/32 (6.25%)  2
Nervous system disorders     
Headache  7/34 (20.59%)  15 6/32 (18.75%)  13
Renal and urinary disorders     
Chromaturia  0/34 (0.00%)  0 3/32 (9.38%)  3
Respiratory, thoracic and mediastinal disorders     
Bronchitis  6/34 (17.65%)  7 4/32 (12.50%)  5
Rhinorrhoea  2/34 (5.88%)  2 3/32 (9.38%)  3
Nasal congestion  1/34 (2.94%)  1 2/32 (6.25%)  2
Oropharyngeal pain  1/34 (2.94%)  1 2/32 (6.25%)  2
Skin and subcutaneous tissue disorders     
Seborrhoeic dermatitis  2/34 (5.88%)  2 1/32 (3.13%)  1
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: Dr Gunnar Buyse
Organization: University Hospital Leuven-Children Hospital
Phone: +32 016343845
EMail: gunnar.buyse@uzleuven.be
Layout table for additonal information
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01027884     History of Changes
Other Study ID Numbers: SNT-III-003
First Submitted: December 8, 2009
First Posted: December 9, 2009
Results First Submitted: June 15, 2015
Results First Posted: October 19, 2015
Last Update Posted: October 19, 2015