Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) (DMD114876)

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: NCT01462292
Recruitment Status : Completed
First Posted : October 31, 2011
Results First Posted : October 16, 2017
Last Update Posted : October 16, 2017
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Muscular Dystrophies
Interventions Drug: GSK2402968 3mg/kg/week
Drug: GSK2402968 6 mg/kg/week
Drug: Placebo to match GSK2402968 3 mg/kg/week
Drug: Placebo to match GSK2402968 6 mg/kg/week
Enrollment 51
Recruitment Details A total of 51 male participants, with Duchenne Muscular Dystrophy were randomized in the study. The study was conducted from 26 October 2011 to 04 November 2013.
Pre-assignment Details  
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 milliliter (mL) vials containing 1 mL sterile solution. The participants in this arm were administered with 3 milligram (mg) per kilogram (kg) GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
Period Title: Overall Study
Started 16 17 18
Completed 16 17 18
Not Completed 0 0 0
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week Total
Hide Arm/Group Description The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL. The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL. Total of all reporting groups
Overall Number of Baseline Participants 16 17 18 51
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 17 participants 18 participants 51 participants
8.0  (1.79) 7.8  (1.91) 7.6  (2.70) 7.8  (2.15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 17 participants 18 participants 51 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
16
 100.0%
17
 100.0%
18
 100.0%
51
 100.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 17 participants 18 participants 51 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
2
  11.1%
2
   3.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   6.3%
1
   5.9%
1
   5.6%
3
   5.9%
White
14
  87.5%
16
  94.1%
15
  83.3%
45
  88.2%
More than one race
1
   6.3%
0
   0.0%
0
   0.0%
1
   2.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Mean Change From Baseline in Muscle Function Using the 6 Minute Walking Distance
Hide Description The participants during this assessment were asked to walk, at their own preferred speed, up and down a fixed distance until they were told to stop after 6 minutes. The participants were warned of the time and were told to stop earlier if they feel unable to continue. The total distance walked within the duration of 6 minutes (or until the participant stopped in case of early termination of the test), was recorded in meters. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT) Population was defined as all participants who were randomized to the study, received at least one dose of study medication and have at least one post-Baseline efficacy assessment
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL.
Overall Number of Participants Analyzed 16 17 18
Least Squares Mean (Standard Error)
Unit of Measure: Meters
-10.98  (10.666) -19.93  (9.964) 16.12  (9.941)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.554
Comments Due to the two comparisons for the two different doses, the type 1 error rate (5% overall) was preserved by utilizing a hierarchical approach, testing the 6mg/kg GSK2402968 dose first
Method Mixed Model Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, centre grouping, baseline and baseline by visit
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -8.946
Confidence Interval (2-Sided) 95%
-39.122 to 21.229
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.069
Comments Due to the two comparisons for the two different doses, the type 1 error rate (5% overall) was preserved by utilising a hierarchical approach, testing the 6mg/kg GSK2402968 dose first
Method Mixed Model Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, centre grouping, baseline and baseline by visit
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 27.099
Confidence Interval (2-Sided) 95%
-2.210 to 56.408
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in Rise From Floor Time at Week 24
Hide Description The rise from floor was assessed, when the participants stood from a standardized supine position as quickly as possible when told to go. Time was recorded with a stopwatch from the initiation of movement until the assumption of upright standing. No aids or orthoses were allowed. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
Overall Number of Participants Analyzed 16 17 18
Least Squares Mean (Standard Error)
Unit of Measure: Seconds
1.12  (0.700) 1.50  (0.662) 1.95  (0.656)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.699
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, centre grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.387
Confidence Interval (2-Sided) 95%
-1.618 to 2.392
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.384
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, centre grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.831
Confidence Interval (2-Sided) 95%
-1.072 to 2.735
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in 4 Stair Climb Ascent/Descent Time at Week 24
Hide Description During this assessment, the participants were asked to ascend and descend four steps. The time for this was recorded with a stopwatch from the initiation of movement until the participant stands on the fourth step, (going up and going down separately). A flight of steps with handrail were used for this test. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
Overall Number of Participants Analyzed 16 17 18
Least Squares Mean (Standard Error)
Unit of Measure: seconds
Ascent time 0.59  (0.308) 0.59  (0.295) -0.22  (0.295)
Descent time 0.60  (0.473) -0.09  (0.442) 0.19  (0.440)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Placebo (combined) Vs GSK2402968 3 mg/kg/week, Ascent Week 24
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.997
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment,centre grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.002
Confidence Interval (2-Sided) 95%
-0.883 to 0.886
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Placebo (combined) Vs GSK2402968 6 mg/kg/week, Ascent, Week 24
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.064
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, centre grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.803
Confidence Interval (2-Sided) 95%
-1.655 to 0.048
Estimation Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Placebo (combined) Vs GSK2402968 3 mg/kg/week, Descent Week 24
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.311
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, centre grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.685
Confidence Interval (2-Sided) 95%
-2.033 to 0.662
Estimation Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Placebo (combined) Vs GSK2402968 6 mg/kg/week, Descent Week 24
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.523
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, centre grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.412
Confidence Interval (2-Sided) 95%
-1.702 to 0.878
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in 10 Meter Walk/Run at Week 24
Hide Description The participants during this assessment were asked to traverse marked 10-meter measured walkway as quickly as he safely can. Time was recorded to one tenth of a second with a stop watch from when his first foot crossed the start line until when the second foot crossed the finish line. How often the participant , touched the wall was to be noted. Care was taken to ensure that the participants were safe when completing this test. The assessor was allowed to walk nearby to provide ‘emergency’ help if needed, but must not support or provide manual assistance for the participant in any way. If the participant was unable to complete the 10-meter walk, the total distance was recorded. The participants were to perform the test in bare feet. No aids or orthoses were allowed. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
Overall Number of Participants Analyzed 16 17 18
Least Squares Mean (Standard Error)
Unit of Measure: Seconds
-0.04  (0.196) 0.52  (0.187) -0.01  (0.185)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.050
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, centre grouping and baseline
Method of Estimation Estimation Parameter Median Difference (Net)
Estimated Value 0.564
Confidence Interval (2-Sided) 95%
0.000 to 1.127
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.890
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, center grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.037
Confidence Interval (2-Sided) 95%
-0.498 to 0.571
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline in Muscle Strength Total Score at Week 24
Hide Description The muscle strength was recorded by handheld myometry using a microFET2 myometer. Upper and lower limb proximal muscles were evaluated including knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors. Total score was calculated by summing up all individual scores. If data for any of the individual muscle strength tests was missing, the total score were set to missing for that visit. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the specified timepoints were analyzed
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
Overall Number of Participants Analyzed 16 17 17
Least Squares Mean (Standard Error)
Unit of Measure: Pounds (lbs)
0.32  (4.633) 2.67  (4.385) 1.14  (4.469)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.723
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model includes terms for Treatment, Centre Grouping and Baseline Muscle Strength Total Score
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.356
Confidence Interval (2-Sided) 95%
-10.936 to 15.648
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.898
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model includes terms for Treatment, Centre Grouping and Baseline Muscle Strength Total Score
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.821
Confidence Interval (2-Sided) 95%
-12.034 to 13.676
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24
Hide Description The muscle strength was recorded by handheld myometry using a microFET2 myometer. Upper and lower limb proximal muscles were evaluated including knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Mean (Standard Deviation)
Unit of Measure: lbs
Knee extensor left Number Analyzed 16 participants 17 participants 18 participants
0.01  (4.574) -1.14  (3.219) 1.02  (3.863)
Knee extensor right Number Analyzed 16 participants 17 participants 18 participants
-0.99  (3.625) -0.88  (3.123) 0.45  (3.235)
Knee flexor left Number Analyzed 16 participants 17 participants 17 participants
0.10  (3.398) 0.37  (2.178) 0.48  (2.211)
Knee flexor right Number Analyzed 16 participants 17 participants 17 participants
0.11  (2.763) 0.05  (2.410) 0.28  (2.039)
Hip flexor left Number Analyzed 16 participants 17 participants 17 participants
-0.53  (2.922) -0.58  (2.848) 1.10  (3.391)
Hip flexor right Number Analyzed 16 participants 17 participants 17 participants
-0.82  (4.190) -0.08  (3.213) 0.57  (3.243)
Elbow flexor left Number Analyzed 16 participants 17 participants 18 participants
0.86  (2.443) 0.56  (2.410) 0.69  (2.273)
Elbow flexor right Number Analyzed 16 participants 17 participants 18 participants
0.47  (2.633) -0.25  (1.737) 0.11  (1.801)
Elbow extensor left Number Analyzed 16 participants 17 participants 18 participants
0.50  (1.344) 0.45  (2.235) 0.56  (2.547)
Elbow extensor right Number Analyzed 16 participants 17 participants 18 participants
0.69  (1.673) 1.11  (2.118) 0.15  (1.715)
Shoulder abductor left Number Analyzed 16 participants 17 participants 18 participants
0.13  (1.423) 0.65  (1.825) 0.31  (1.710)
Shoulder abductor right Number Analyzed 16 participants 17 participants 18 participants
0.29  (1.588) 0.26  (2.150) 0.69  (2.106)
7.Secondary Outcome
Title Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score
Hide Description The NSAA was a functional scale devised from Hammersmith Scale of Motor Ability specifically for use in ambulant children with DMD. It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). The scale assessed activities that required for ambulatory activity and included items that were rarely achieved in untreated DMD (jump, hop, raise head) as well as items that are known to progressively deteriorate over time (stand from a chair, walk). A standardized manual is available within the SPM with specific instructions for grading. Video snaps used in training program to ensure evaluator reliability. The total score ranged from 0-34 where the highest score of 34 implies absence of symptoms and lower score implies more severe symptoms. Change from Baseline, was defined as the post-randomization value minus the baseline value. Baseline was defined as Week 0.
Time Frame Baselie (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Least Squares Mean (Standard Error)
Unit of Measure: Scores on scale
-0.6  (0.84) -1.1  (0.80) -0.8  (0.79)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.684
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, center grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.49
Confidence Interval (2-Sided) 95%
-2.90 to 1.92
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6 mg
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.873
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method ANCOVA
Comments Model included terms for treatment, center grouping and baseline
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.18
Confidence Interval (2-Sided) 95%
-2.49 to 2.12
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Number of Participants With Accidental Falls During 6 Minute Walk Distance Test
Hide Description The participants during the 6 minute walk distance were asked to walk, at their own preferred speed, up and down a fixed distance until they were told to stop after 6 minutes. The participants were warned of the time and were told to stop earlier if they feel unable to continue. The total distance walked within the duration of 6 minutes (or until the participant stopped in case of early termination of the test), was recorded in metersThe number of accident falls during the 6 minute walk distance were reported. Data is reported for the number of participants with accidental falls of 0, 1 and 2.
Time Frame Baseline (Week 0), Week 24, Week 36 and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the specified timepoints were analyzed
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Measure Type: Number
Unit of Measure: Participants
Baseline, 0 fall Number Analyzed 16 participants 17 participants 18 participants
16 16 17
Baseline, 1 fall Number Analyzed 16 participants 17 participants 18 participants
0 1 1
Week 12, 0 fall Number Analyzed 16 participants 17 participants 18 participants
15 17 17
Week 12, 1 fall Number Analyzed 16 participants 17 participants 18 participants
1 0 1
Week 24, 0 fall Number Analyzed 16 participants 17 participants 18 participants
16 16 16
Week 24, 1 fall Number Analyzed 16 participants 17 participants 18 participants
0 1 2
Week 36, 0 fall Number Analyzed 14 participants 14 participants 14 participants
14 13 14
Week 36, 2 falls Number Analyzed 14 participants 14 participants 14 participants
0 1 0
Week 48, 0 fall Number Analyzed 11 participants 14 participants 13 participants
10 14 13
Week 48, 3 falls Number Analyzed 11 participants 14 participants 13 participants
1 0 0
9.Secondary Outcome
Title Change From Baseline in Creatinine Kinase Serum Concentrations
Hide Description Creatine kinase (CK) is a muscle-specific enzyme; its level in plasma is considered to reflect the extent of muscle damage. In the blood samples drawn to this purpose, the plasma level of CK was measured. Change from Baseline, was defined as the post-randomization value minus the baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the specified time points were analyzed.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Least Squares Mean (Standard Error)
Unit of Measure: International units per liter
Week 24 Number Analyzed 16 participants 17 participants 17 participants
-3303.5  (1224.65) -4196.4  (1171.20) -4609.0  (1170.75)
Week 48 Number Analyzed 15 participants 17 participants 18 participants
-2783.1  (1248.65) -4838.0  (1170.75) -2615.7  (1149.44)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg, Week 24
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.610
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method Mixed Model Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, center grouping, baseline and baseline by visit
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -892.88
Confidence Interval (2-Sided) 95%
-4391.10 to 2605.35
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 3 mg, Week 48
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.248
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method Mixed Model Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, center grouping, baseline and baseline by visit
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -2054.86
Confidence Interval (2-Sided) 95%
-5587.33 to 1477.60
Estimation Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6 mg, Week 24
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.439
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method Mixed Model Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, center grouping, baseline and baseline by visit
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1305.46
Confidence Interval (2-Sided) 95%
-4668.41 to 2057.48
Estimation Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Treatment difference: Placebo Vs GSK2402968 6 mg, Week 48
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.921
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method Mixed Model Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, center grouping, baseline and baseline by visit
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 167.39
Confidence Interval (2-Sided) 95%
-3208.98 to 3543.77
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Change From Baseline in Forced Expiratory Volume in the First Second of Exhalation (FEV1) at Week 24
Hide Description FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated timepoints were used for analysis
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 16 15
Mean (Standard Deviation)
Unit of Measure: Litres
0.054  (0.2445) -0.009  (0.2316) 0.015  (0.1647)
11.Secondary Outcome
Title Change From Baseline in Forced Vital Capacity (FVC) at Week 24
Hide Description FVC is defined as the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. Change from Baseline, was defined as the post-randomization value minus the baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the indicated timepoints were used for analysis.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 16 15
Mean (Standard Deviation)
Unit of Measure: Litres
0.063  (0.2670) 0.080  (0.2064) 0.051  (0.1419)
12.Secondary Outcome
Title Change From Baseline in Peak Cough Flow at Week 24
Hide Description The peak cough flow was conducted using a spirometer. Change from Baseline, was defined as the post-randomization value minus the Baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the indicated timepoints were used for analysis
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 15 15 17
Mean (Standard Deviation)
Unit of Measure: Litres per minute
16.5  (19.03) 18.2  (24.19) 18.0  (28.09)
13.Secondary Outcome
Title Change From Baseline in Peak Expiratory Flow at Week 24
Hide Description The peak expiratory flow is a measure of the amount of air that can be pushed through the airways in a single rapid exhalation. The peak expiratory flow was measured using spirometry. Change from Baseline, was defined as the post-randomization value minus the baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the specified time points were analyzed
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 16 17
Mean (Standard Deviation)
Unit of Measure: Litres per minute
18.9  (35.89) 2.5  (35.98) 6.6  (34.68)
14.Secondary Outcome
Title Change From Baseline in Sniff Pressure Test at Week 24
Hide Description This was one of the pulmonary function test which was a non-invasive procedure. It measured the inspiratory muscle strength by transdiaphragmatic (Pdi) and esophageal pressures (Pes) generated during volitional and nonvolitional maneuvers. Change from Baseline, was defined as the post-randomization value minus the baseline value. Baseline was defined as Week 0.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the specified time points were analyzed
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 15 16
Mean (Standard Deviation)
Unit of Measure: Centimeter of water
3.46  (12.988) -1.47  (10.155) 2.75  (16.838)
15.Secondary Outcome
Title Number of Participants With Change From Baseline in Dystrophin Expression at Week 24 by Immunofluorescence Assay (IFA)
Hide Description A muscle biopsy from the tibialis anterior muscle was taken to assess the expression of dystrophin. The muscle biopsy samples were collected by open biopsy or with the conchotome method according to standard hospital procedures for obtaining muscle biopsies from children. The minimum amount of muscle tissue required is a small piece of muscle of at least 0.5 x 0.5 x 0.5 centimeters. The muscle tissue was immediately frozen in liquid nitrogen-cooled 2-methylbutane and stored at -80°Celsius (C) or -70°C till shipment. In case of DMD participants , there is defect in the dystrophin producing gene or absence. Data for number of participants with change from baseline in dystrophin expression, was diagnosed using IFA and was categorized as strong increase, increase, and no change, decrease.
Time Frame Baseline (Week 0) and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the specified timepoints were analyzed.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 12 11 13
Measure Type: Count of Participants
Unit of Measure: Participants
Strong increase
5
  41.7%
3
  27.3%
0
   0.0%
Increase
2
  16.7%
2
  18.2%
1
   7.7%
Decrease
2
  16.7%
3
  27.3%
4
  30.8%
No change
3
  25.0%
3
  27.3%
8
  61.5%
16.Secondary Outcome
Title Number of Clinician Global Impression of Improvement (CGI-I) Responders
Hide Description Single item question designed to provide a brief, stand-alone assessment of the clinician’s view of the participant’s global functioning after initiating a study medication, compared to their global functioning just prior to initiating treatment. Evaluated by an expert physician or evaluator familiar with DMD and who could make an expert clinical global judgement about severity of illness across various time points within context of clinical experience. The CGI-I reflects the clinician’s judgment about the total picture of the participant : the illness severity, the level of distress and other aspects of impairment, and impact of illness on functioning. The CGI-I is rated without regard to clinician’s belief that any clinical changes are or are not due to medication and without consideration of etiology of symptoms. It is measured on 7-point Likert scale (1 = ‘very much improved’, 2 = ‘much improved’, 4 = ‘no change’, 5 = ‘minimally worse’, 6 = ‘much worse’, 7 = ‘very much worse’).
Time Frame Week 24 and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the specified time points were analyzed.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
Week 24 Number Analyzed 14 participants 17 participants 18 participants
2
  14.3%
1
   5.9%
2
  11.1%
Week 48 Number Analyzed 14 participants 17 participants 16 participants
1
   7.1%
0
   0.0%
2
  12.5%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 3 mg/kg/Week
Comments Placebo (combined), GSK2402968 3 mg/kg/week
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.541
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method Regression, Logistic
Comments Model includes terms for Treatment and Centre Grouping
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.41
Confidence Interval (2-Sided) 95%
0.02 to 7.01
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo (Combined), GSK2402968 6 mg/kg/Week
Comments Placebo (combined), GSK2402968 6 mg/kg/week
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.417
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance
Method Regression, Logistic
Comments Model includes terms for Treatment and Centre Grouping
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.36
Confidence Interval (2-Sided) 95%
0.03 to 4.27
Estimation Comments [Not Specified]
17.Secondary Outcome
Title Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period
Hide Description This was conducted by the family or caregiver. This helped to document the observed changes in the participant’s functional outcome like the day to day activities; general health, mobility, and other general daily activities. The data for Week 24 has been reported as improved, not improved and not applicable.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at that particular time points were analyzed.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
Ability to walk 50 feet, improved
4
  25.0%
4
  23.5%
8
  44.4%
Ability to walk 50 feet, not improved
12
  75.0%
13
  76.5%
10
  55.6%
Ability to walk 50 feet, not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Rate your child’s condition, improved
5
  31.3%
6
  35.3%
6
  33.3%
Rate your child’s condition, not improved
11
  68.8%
11
  64.7%
12
  66.7%
Walk 15-20 mins without tired,improved
5
  31.3%
5
  29.4%
6
  33.3%
Walk 15-20 mins without tired,not improved
11
  68.8%
11
  64.7%
12
  66.7%
Walk 15-20 mins without tired,not applicable
0
   0.0%
1
   5.9%
0
   0.0%
Able to stand 10 mins without tired, improved
3
  18.8%
4
  23.5%
4
  22.2%
Able to stand 10 mins without tired,not improved
13
  81.3%
13
  76.5%
14
  77.8%
Able to stand 10 mins without tired,not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Able to step up on stool, improved
3
  18.8%
5
  29.4%
5
  27.8%
Able to step up on stool, not improved
13
  81.3%
11
  64.7%
13
  72.2%
Able to step up on stool, not applicable
0
   0.0%
1
   5.9%
0
   0.0%
Able to walk up/down a slope, improved
4
  25.0%
5
  29.4%
6
  33.3%
Able to walk up/down a slope, not improved
11
  68.8%
12
  70.6%
12
  66.7%
Able to walk up/down a slope, not applicable
1
   6.3%
0
   0.0%
0
   0.0%
Able to climb 3-4 steps, improved
2
  12.5%
5
  29.4%
5
  27.8%
Able to climb 3-4 steps, not improved
14
  87.5%
12
  70.6%
13
  72.2%
Able to climb 3-4 steps, not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Able to climb 10-15 steps, improved
2
  12.5%
2
  11.8%
3
  16.7%
Able to climb 10-15 steps, not improved
14
  87.5%
14
  82.4%
14
  77.8%
Able to climb 10-15 steps, not applicable
0
   0.0%
1
   5.9%
1
   5.6%
Able to walk on uneven surfaces, improved
4
  25.0%
3
  17.6%
5
  27.8%
Able to walk on uneven surfaces, not improved
12
  75.0%
14
  82.4%
13
  72.2%
Able to walk on uneven surfaces, not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Able to walk without fall, improved
6
  37.5%
5
  29.4%
8
  44.4%
Able to walk without fall, not improved
10
  62.5%
12
  70.6%
10
  55.6%
Able to walk without fall, not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Participate in physical activities, improved
4
  25.0%
3
  17.6%
5
  27.8%
Participate in physical activities, not improved
12
  75.0%
13
  76.5%
13
  72.2%
Participate in physical activities, not applicable
0
   0.0%
1
   5.9%
0
   0.0%
Able to go out with friends/family, improved
6
  37.5%
5
  29.4%
5
  27.8%
Able to go out with friends/family, not improved
10
  62.5%
12
  70.6%
12
  66.7%
Able to go out with friends/family, not applicable
0
   0.0%
0
   0.0%
1
   5.6%
Able to throw and catch ball, improved
3
  18.8%
2
  11.8%
6
  33.3%
Able to throw and catch ball, not improved
13
  81.3%
15
  88.2%
12
  66.7%
Able to throw and catch ball, not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Able to run and kick a ball, improved
4
  25.0%
1
   5.9%
6
  33.3%
Able to run and kick a ball, not improved
12
  75.0%
15
  88.2%
12
  66.7%
Able to run and kick a ball, not applicable
0
   0.0%
1
   5.9%
0
   0.0%
Able to get in and out of bed, improved
4
  25.0%
4
  23.5%
6
  33.3%
Able to get in and out of bed, not improved
12
  75.0%
13
  76.5%
11
  61.1%
Able to get in and out of bed, not applicable
0
   0.0%
0
   0.0%
1
   5.6%
Able to get in and out of car, improved
4
  25.0%
3
  17.6%
5
  27.8%
Able to get in and out of car, not improved
12
  75.0%
14
  82.4%
12
  66.7%
Able to get in and out of car, not applicable
0
   0.0%
0
   0.0%
1
   5.6%
Able to pick small item from table, improved
1
   6.3%
2
  11.8%
4
  22.2%
Able to pick small item from table, not improved
15
  93.8%
15
  88.2%
13
  72.2%
Able to pick small item from table, not applicable
0
   0.0%
0
   0.0%
1
   5.6%
Able to pick large item from table, improved
2
  12.5%
3
  17.6%
5
  27.8%
Able to pick large item from table, not improved
14
  87.5%
14
  82.4%
13
  72.2%
Able to play with toys, improved
1
   6.3%
4
  23.5%
3
  16.7%
Able to play with toys, not improved
14
  87.5%
13
  76.5%
13
  72.2%
Able to play with toys, not applicable
1
   6.3%
0
   0.0%
2
  11.1%
Able to play board or video games, improved
4
  25.0%
4
  23.5%
7
  38.9%
Able to play board or video games, not improved
12
  75.0%
13
  76.5%
11
  61.1%
Able to play board or video games, not applicable
0
   0.0%
0
   0.0%
0
   0.0%
Able to work on computer, improved
3
  18.8%
2
  11.8%
4
  22.2%
Able to work on computer, not improved
13
  81.3%
15
  88.2%
12
  66.7%
Able to work on computer, not applicable
0
   0.0%
0
   0.0%
2
  11.1%
Able to squeeze out toothpaste, improved
1
   6.3%
2
  11.8%
3
  16.7%
Able to squeeze out toothpaste, not improved
15
  93.8%
12
  70.6%
14
  77.8%
Able to squeeze out toothpaste, not applicable
0
   0.0%
3
  17.6%
1
   5.6%
Able to brush teeth, improved
2
  12.5%
2
  11.8%
4
  22.2%
Able to brush teeth, not improved
14
  87.5%
15
  88.2%
13
  72.2%
Able to brush teeth, not applicable
0
   0.0%
0
   0.0%
1
   5.6%
Pour milk or juice from in a cup , improved
2
  12.5%
0
   0.0%
5
  27.8%
Pour milk or juice from in a cup, not improved
14
  87.5%
16
  94.1%
11
  61.1%
Pour milk or juice from in a cup, not applicable
0
   0.0%
1
   5.9%
2
  11.1%
Able to drink from cup without straw, improved
2
  12.5%
2
  11.8%
4
  22.2%
Able to drink from cup without straw, not improved
14
  87.5%
15
  88.2%
13
  72.2%
Drink from cup without straw, not applicable
0
   0.0%
0
   0.0%
1
   5.6%
Able to button shirt or jacket, improved
2
  12.5%
0
   0.0%
6
  33.3%
Able to button shirt or jacket, not improved
14
  87.5%
16
  94.1%
10
  55.6%
Able to button shirt or jacket, not applicable
0
   0.0%
1
   5.9%
2
  11.1%
Able to zip jacket or pants, improved
1
   6.3%
1
   5.9%
4
  22.2%
Able to zip jacket or pants, not improved
14
  87.5%
16
  94.1%
13
  72.2%
Able to zip jacket or pants, not applicable
1
   6.3%
0
   0.0%
1
   5.6%
Childs need for orthotics, improved
0
   0.0%
0
   0.0%
0
   0.0%
Childs need for orthotics, not improved
9
  56.3%
10
  58.8%
5
  27.8%
Childs need for orthotics, not applicable
7
  43.8%
7
  41.2%
13
  72.2%
Childs need for wheelchair, improved
1
   6.3%
0
   0.0%
1
   5.6%
Childs need for wheelchair, not improved
6
  37.5%
10
  58.8%
5
  27.8%
Childs need for wheelchair, not applicable
9
  56.3%
7
  41.2%
12
  66.7%
18.Secondary Outcome
Title Assessment of Functional Outcome by : Physician Assessment of Daily Living
Hide Description This was conducted by the physician, which helped to assess and document observed changes in the participant by the physician reported by the participant or his family or caregiver. This was reported as any worsening and any improvement up to week 24.
Time Frame Week 24 and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the specified time points were analyzed.
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description:
The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution
The participants in this arm were administered with 3 mg per kilogram kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg per mL
Overall Number of Participants Analyzed 16 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
Week 12, Any improvement Number Analyzed 16 participants 16 participants 16 participants
9
  56.3%
6
  37.5%
12
  75.0%
Week 12, Any worsening Number Analyzed 16 participants 16 participants 16 participants
2
  12.5%
1
   6.3%
0
   0.0%
Week 24, Any improvement Number Analyzed 15 participants 17 participants 18 participants
10
  66.7%
8
  47.1%
13
  72.2%
Week 24, Any worsening Number Analyzed 15 participants 17 participants 18 participants
5
  33.3%
3
  17.6%
3
  16.7%
Week 48, Any improvement Number Analyzed 10 participants 14 participants 9 participants
5
  50.0%
5
  35.7%
7
  77.8%
Week 48, Any worsening Number Analyzed 10 participants 14 participants 9 participants
6
  60.0%
6
  42.9%
3
  33.3%
Anytime during study, Any improvement Number Analyzed 16 participants 17 participants 18 participants
12
  75.0%
11
  64.7%
17
  94.4%
Anytime during study, Any worsening Number Analyzed 16 participants 17 participants 18 participants
11
  68.8%
8
  47.1%
6
  33.3%
Time Frame Up to Week 48
Adverse Event Reporting Description Safety Population was defined as all participants who received at least one dose of study medication.
 
Arm/Group Title Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Hide Arm/Group Description The participants in this arm were administered matching placebo subcutaneously for 24 Weeks. It was provided as 3 mL vials containing 1 mL sterile solution The participants in this arm were administered with 3 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL The participants in this arm were administered with 6 mg per kg GSK2402968, subcutaneously for 24 weeks. It was provided as 3 mL vials containing 1 mL sterile solution. The concentration of drisapersen solution was 200 mg/mL
All-Cause Mortality
Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/16 (0.00%)      0/17 (0.00%)      0/18 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/16 (6.25%)      0/17 (0.00%)      0/18 (0.00%)    
Infections and infestations       
Wound infection staphylococcal  1  1/16 (6.25%)  0/17 (0.00%)  0/18 (0.00%) 
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo (Combined) GSK2402968 3 mg/kg/Week GSK2402968 6 mg/kg/Week
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/16 (81.25%)      16/17 (94.12%)      17/18 (94.44%)    
Cardiac disorders       
Systolic dysfunction  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Ear and labyrinth disorders       
Tinnitus  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Eye disorders       
Conjunctivitis  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Gastrointestinal disorders       
Vomiting  1  4/16 (25.00%)  4 1/17 (5.88%)  2 3/18 (16.67%)  4
Diarrhoea  1  2/16 (12.50%)  2 1/17 (5.88%)  1 3/18 (16.67%)  5
Nausea  1  3/16 (18.75%)  3 1/17 (5.88%)  1 1/18 (5.56%)  1
Abdominal discomfort  1  2/16 (12.50%)  4 0/17 (0.00%)  0 2/18 (11.11%)  2
Constipation  1  1/16 (6.25%)  1 1/17 (5.88%)  1 1/18 (5.56%)  1
Abdominal pain upper  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
Food poisoning  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  2
Gastrooesophageal reflux disease  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Lip swelling  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Tongue disorder  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
General disorders       
Injection site erythema  1  1/16 (6.25%)  1 10/17 (58.82%)  63 9/18 (50.00%)  97
Injection site discolouration  1  1/16 (6.25%)  1 5/17 (29.41%)  19 5/18 (27.78%)  37
Pyrexia  1  2/16 (12.50%)  2 1/17 (5.88%)  1 5/18 (27.78%)  7
Injection site bruising  1  1/16 (6.25%)  1 2/17 (11.76%)  3 3/18 (16.67%)  5
Injection site pruritus  1  1/16 (6.25%)  1 2/17 (11.76%)  12 1/18 (5.56%)  4
Injection site haematoma  1  2/16 (12.50%)  2 0/17 (0.00%)  0 0/18 (0.00%)  0
Injection site pain  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
Injection site swelling  1  0/16 (0.00%)  0 1/17 (5.88%)  1 1/18 (5.56%)  1
Vessel puncture site bruise  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
Application site bruise  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Infusion site haematoma  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Injection site haemorrhage  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Injection site oedema  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  3
Injection site reaction  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Non-cardiac chest pain  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Pain  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Vessel puncture site pain  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Immune system disorders       
Hypersensitivity  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Infections and infestations       
Nasopharyngitis  1  2/16 (12.50%)  3 4/17 (23.53%)  5 3/18 (16.67%)  5
Upper respiratory tract infection  1  3/16 (18.75%)  3 3/17 (17.65%)  4 0/18 (0.00%)  0
Gastroenteritis viral  1  2/16 (12.50%)  2 1/17 (5.88%)  1 0/18 (0.00%)  0
Otitis media  1  1/16 (6.25%)  1 2/17 (11.76%)  2 0/18 (0.00%)  0
Influenza  1  0/16 (0.00%)  0 0/17 (0.00%)  0 2/18 (11.11%)  2
Ear infection  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Oral herpes  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Paronychia  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Pharyngitis streptococcal  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Pneumonia  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Sinusitis  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Stitch abscess  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Viral infection  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Viral upper respiratory tract infection  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Injury, poisoning and procedural complications       
Procedural pain  1  3/16 (18.75%)  7 1/17 (5.88%)  1 4/18 (22.22%)  6
Contusion  1  1/16 (6.25%)  1 0/17 (0.00%)  0 2/18 (11.11%)  2
Fall  1  1/16 (6.25%)  1 0/17 (0.00%)  0 2/18 (11.11%)  3
Ligament sprain  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
Postoperative wound complication  1  0/16 (0.00%)  0 0/17 (0.00%)  0 2/18 (11.11%)  2
Arthropod bite  1  1/16 (6.25%)  4 0/17 (0.00%)  0 0/18 (0.00%)  0
Excoriation  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  3
Hand fracture  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Laceration  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Post procedural discomfort  1  1/16 (6.25%)  2 0/17 (0.00%)  0 0/18 (0.00%)  0
Thermal burn  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Wrist fracture  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Investigations       
Alanine aminotransferase increased  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
International normalised ratio increased  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
Protein urine present  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  1
Activated partial thromboplastin time prolonged  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Aspartate aminotransferase increased  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Blood fibrinogen decreased  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Blood immunoglobulin G decreased  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Creatinine renal clearance decreased  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
ECG signs of ventricular hypertrophy  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Electrocardiogram QT prolonged  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Fibrin D dimer increased  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Red blood cells urine  1  0/16 (0.00%)  0 1/17 (5.88%)  2 0/18 (0.00%)  0
Reticulocyte count increased  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Urine analysis abnormal  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Vitamin D decreased  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Metabolism and nutrition disorders       
Decreased appetite  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Obesity  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Pain in extremity  1  4/16 (25.00%)  7 1/17 (5.88%)  1 0/18 (0.00%)  0
Arthralgia  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Flank pain  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Muscle hypertrophy  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Muscle spasms  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Muscular weakness  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Nervous system disorders       
Headache  1  4/16 (25.00%)  24 3/17 (17.65%)  7 2/18 (11.11%)  7
Dizziness  1  2/16 (12.50%)  2 0/17 (0.00%)  0 0/18 (0.00%)  0
Balance disorder  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Burning sensation  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Paraesthesia  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Tongue biting  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Psychiatric disorders       
Anxiety  1  2/16 (12.50%)  2 1/17 (5.88%)  1 0/18 (0.00%)  0
Abnormal behaviour  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Agitation  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Autism spectrum disorder  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Sleep disorder  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Renal and urinary disorders       
Proteinuria  1  2/16 (12.50%)  2 1/17 (5.88%)  1 3/18 (16.67%)  3
Chromaturia  1  2/16 (12.50%)  2 1/17 (5.88%)  1 1/18 (5.56%)  1
Renal impairment  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  2
Haematuria  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Hydronephrosis  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Nephrolithiasis  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Nasal congestion  1  3/16 (18.75%)  3 2/17 (11.76%)  2 4/18 (22.22%)  5
Cough  1  3/16 (18.75%)  3 1/17 (5.88%)  1 2/18 (11.11%)  2
Rhinorrhoea  1  0/16 (0.00%)  0 1/17 (5.88%)  1 3/18 (16.67%)  5
Oropharyngeal pain  1  0/16 (0.00%)  0 1/17 (5.88%)  1 1/18 (5.56%)  1
Epistaxis  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Pulmonary congestion  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Upper respiratory tract congestion  1  0/16 (0.00%)  0 1/17 (5.88%)  1 0/18 (0.00%)  0
Upper-airway cough syndrome  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Skin and subcutaneous tissue disorders       
Dermatitis contact  1  0/16 (0.00%)  0 2/17 (11.76%)  3 1/18 (5.56%)  1
Rash pruritic  1  2/16 (12.50%)  6 0/17 (0.00%)  0 0/18 (0.00%)  0
Urticaria  1  1/16 (6.25%)  1 0/17 (0.00%)  0 1/18 (5.56%)  2
Onychoclasis  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Onychomadesis  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  1
Rash  1  0/16 (0.00%)  0 1/17 (5.88%)  2 0/18 (0.00%)  0
Skin discolouration  1  0/16 (0.00%)  0 0/17 (0.00%)  0 1/18 (5.56%)  3
Skin lesion  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
Vascular disorders       
Flushing  1  1/16 (6.25%)  1 0/17 (0.00%)  0 0/18 (0.00%)  0
1
Term from vocabulary, MedDRA 16.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
GSK agreements may vary with individual investigators, but will not prohibit any investigator from publishing. GSK supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial.
Results Point of Contact
Name/Title: GSK Response Center
Organization: GlaxoSmithKline
Phone: 866-435-7343
Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01462292     History of Changes
Other Study ID Numbers: 114876
First Submitted: October 3, 2011
First Posted: October 31, 2011
Results First Submitted: August 3, 2017
Results First Posted: October 16, 2017
Last Update Posted: October 16, 2017