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A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD114044)

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: NCT01254019
Recruitment Status : Completed
First Posted : December 6, 2010
Results First Posted : January 28, 2019
Last Update Posted : January 28, 2019
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Muscular Dystrophies
Intervention Drug: GSK2402968 6mg/kg/week
Enrollment 186
Recruitment Details This study was conducted across 44 centers in 19 countries from 02 December 2010 to 28 June 2013.
Pre-assignment Details A total of 186 participants were randomized which included males with a maximum age of 16 years, no adults were included in this study.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period. Participants received single dose of GSK2402968 6 milligrams per kilogram per week (mg/kg/week) subcutaneous injection preferably in the morning over the 48 week treatment period.
Period Title: Overall Study
Started 61 125
Completed 60 121
Not Completed 1 4
Reason Not Completed
Adverse Event             0             2
Protocol Violation             1             0
Withdrawal by Subject             0             2
Arm/Group Title Placebo GSK2402968 6mg/kg/Week Total
Hide Arm/Group Description Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period. Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period. Total of all reporting groups
Overall Number of Baseline Participants 61 125 186
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 61 participants 125 participants 186 participants
8.0  (2.37) 8.3  (2.43) 8.2  (2.41)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 61 participants 125 participants 186 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
61
 100.0%
125
 100.0%
186
 100.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 61 participants 125 participants 186 participants
African American/African Heritage
1
   1.6%
0
   0.0%
1
   0.5%
Asian - Central/South Asian Heritage
1
   1.6%
3
   2.4%
4
   2.2%
Asian - East Asian Heritage
3
   4.9%
6
   4.8%
9
   4.8%
Asian - Japanese Heritage
5
   8.2%
9
   7.2%
14
   7.5%
Asian - South East Asian Heritage
0
   0.0%
2
   1.6%
2
   1.1%
White - Arabic/North African Heritage
4
   6.6%
5
   4.0%
9
   4.8%
White - White/Caucasian/European Heritage
46
  75.4%
95
  76.0%
141
  75.8%
White - Mixed Race
0
   0.0%
1
   0.8%
1
   0.5%
Mixed Race
1
   1.6%
4
   3.2%
5
   2.7%
1.Primary Outcome
Title Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48
Hide Description During the 6MWD, participants 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 that they may stop earlier if they feel unable to continue. The total distance walked within 6 minutes (or until the participant stopped in case of early termination of the test), the 6MWD, was recorded in meters as well as any falls. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat (ITT) Population comprised all participants who received at least one dose of study medication and for whom at least one post-Baseline efficacy assessment was available. Only those participants available at the specified time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 59 117
Least Squares Mean (Standard Error)
Unit of Measure: Meters
-52.65  (10.423) -42.32  (7.378)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.415
Comments Statistical significance was assessed at the 5% level.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 10.334
Confidence Interval (2-Sided) 95%
-14.645 to 35.312
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in the Linearized North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Hide Description The NSAA is a functional scale devised from the Hammersmith Scale of Motor Ability specifically for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was achieved by adding the responses of all activities, ranging from 0 to 34, with a score of 34 implying normal function and lower score implying more severe symptoms. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48. A positive change from Baseline indicated improvement.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 58 117
Least Squares Mean (Standard Error)
Unit of Measure: Scores on a scale
-6.7  (1.43) -7.2  (1.01)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.757
Comments Statistical significance (SS) was only to be assessed if a statistically significant difference was observed for the primary and any secondary endpoints higher in the pre-defined hierarchy. Hence conclusions with regards to SS should not be made.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.53
Confidence Interval (2-Sided) 95%
-3.95 to 2.88
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in the 4 Stair Climb (Ascent) Velocity at Week 48
Hide Description The participant was asked to ascend four steps. Time was recorded with a stopwatch from the initiation of movement until the participant stood on the fourth step. A flight of steps with handrail was used for this test. Number of stairs ascended per second was calculated as 4 divided by the time to ascend 4 complete stairs. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 55 111
Least Squares Mean (Standard Error)
Unit of Measure: Stairs per second
-0.12  (0.049) -0.14  (0.035)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.718
Comments SS was only to be assessed if a statistically significant difference was observed for the primary and any secondary endpoints higher in the pre-defined hierarchy. Hence conclusions with regards to SS should not be made.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.021
Confidence Interval (2-Sided) 95%
-0.137 to 0.095
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in the 10-meter Walk/Run Velocity at Week 48
Hide Description The participant was instructed to perform the test bare foot. No aids or orthoses were allowed. The participant was asked to traverse a marked 10-meter measured walkway as quickly as he safely could. 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. If the wall was touched, it was noted how often. Care was taken to ensure that the participant was safe when completing this test. The assessor walked nearby to provide emergency help if needed, but did not support or provide manual assistance to the participant in any way. If the participant could not complete the 10-meter walk, the total distance was recorded. 10 minute walk/run speed was equal to 10 divided by time taken to complete 10 minute walk/run. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 58 117
Least Squares Mean (Standard Error)
Unit of Measure: Meters per second
-0.20  (0.050) -0.21  (0.035)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.881
Comments SS was only to be assessed if a statistically significant difference was observed for the primary and any secondary endpoints higher in the pre-defined hierarchy. Hence conclusions with regards to SS should not be made.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.009
Confidence Interval (2-Sided) 95%
-0.129 to 0.111
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline in the Timed Function Test Rise From Floor at Week 48
Hide Description The participant 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 are allowed. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 44 91
Least Squares Mean (Standard Error)
Unit of Measure: Seconds
7.48  (2.080) 6.36  (1.463)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.658
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.115
Confidence Interval (2-Sided) 95%
-6.097 to 3.866
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline in the 4 Stair Climb (Descent) Velocity at Week 48
Hide Description The participant was asked to descend four steps. Time was recorded with a stopwatch from the initiation of movement until the participant stood on the fourth step. A flight of steps with handrail was used for this test. Number of stairs descended per second was calculated as 4 divided by the time to descend 4 complete stairs. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 55 109
Least Squares Mean (Standard Error)
Unit of Measure: Stairs per second
-0.15  (0.052) -0.11  (0.037)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.513
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.041
Confidence Interval (2-Sided) 95%
-0.082 to 0.164
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Change From Baseline in Muscle Strength (Total Score) at Week 48
Hide Description Muscle strength was recorded by handheld myometry using a micro force evaluation testing 2 (FET2) myometer. Upper and lower limb proximal muscles were evaluated including knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors. The muscle strength total score (pounds) was the sum of the 12 individual muscle strength tests. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 58 118
Least Squares Mean (Standard Error)
Unit of Measure: Pounds
-1.21  (2.729) -2.18  (1.926)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.769
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.965
Confidence Interval (2-Sided) 95%
-7.446 to 5.516
Estimation Comments [Not Specified]
8.Secondary Outcome
Title Kaplan-Meier Estimates for Time to Loss of Ambulation
Hide Description All participants were ambulant when entered into the study; however they could have become non-ambulant at some time during the study. The date was recorded and the variable time to loss of ambulation was calculated as: time to loss of ambulation = date of loss of ambulation – date of first dose. Median and interquartile range i.e. 1st and 3rd quartile is presented.
Time Frame Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Median (Inter-Quartile Range)
Unit of Measure: Days
NA [1] 
(NA to NA)
NA [2] 
(390.0 to NA)
[1]
Median, upper limit and lower limit was not reached due to insufficient number of participants with events.
[2]
Median and upper limit was not reached due to insufficient number of participants with events.
9.Secondary Outcome
Title Number of Participants Who Experienced Accidental Falls During 6MWD Assessments at Week 48
Hide Description The number of accidental falls occurring during the 6MWD were counted. Data has been presented for the number of participants who experienced accidental falls (from 0 to 1) during the 6MWD assessment.
Time Frame Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants with data available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 53 103
Measure Type: Count of Participants
Unit of Measure: Participants
Number of falls=0
48
  90.6%
102
  99.0%
Number of falls=1
5
   9.4%
1
   1.0%
10.Secondary Outcome
Title Change From Baseline in Creatine Kinase Serum Concentrations at Week 48
Hide Description Creatine kinase is a muscle-specific enzyme; its level in serum is considered to reflect the extent of muscle damage. In the blood samples drawn to this purpose, the serum level of creatine kinase were measured. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo 1 mL sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 60 118
Least Squares Mean (Standard Error)
Unit of Measure: International units per liter
-1228.5  (500.59) -5273.5  (359.05)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.000
Comments No adjustment for multiplicity was made, so p-values should not be used to make conclusions with regards to statistical significance.
Method Mixed Effect Model for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -4044.99
Confidence Interval (2-Sided) 95%
-5232.21 to -2857.77
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Change From Baseline in Pulmonary Function Test Forced Vital Capacity (FVC) and Forced Expiratory Volume in 1 Second (FEV1) at Week 48
Hide Description The FEV1 is the volume of air forcefully exhaled in 1 second, whereas the FVC is the volume of air that can be maximally forcefully exhaled using non-invasive spirometry was conducted to determine actual and percentage values for FVC and FEV1. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the specified time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Mean (Standard Deviation)
Unit of Measure: Liters
FVC at Week 48 Number Analyzed 58 participants 121 participants
0.118  (0.1847) 0.087  (0.2337)
FEV1 at Week 48 Number Analyzed 55 participants 116 participants
0.126  (0.2530) 0.049  (0.2997)
12.Secondary Outcome
Title Number of Participants With Identified Mutation: DMD Exon 51 Skip (Upon Muscle Biopsies) at Week 48
Hide Description Biopsies were taken from their tibialis anterior muscle and few were taken from quadriceps. Total muscle ribonucleic acid (RNA) was isolated from muscle tissue sections and was analyzed by reverse transcriptase polymer chain reaction (RT-PCR). RT-PCR analysis focused on the area flanking the targeted exon 51 was performed to detect specific exon 51 skipping in muscle. Depending on the participants mutation different sets of DMD-gene specific RT and PCR primers were used. Sequence analysis was performed on isolated PCR products to confirm specific exon 51 skip band detection.
Time Frame Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants with data available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 59 119
Measure Type: Count of Participants
Unit of Measure: Participants
DMD Exon 51 skip, Band detected
48
  81.4%
106
  89.1%
DMD Exon 51 skip, No Band detected
9
  15.3%
9
   7.6%
DMD Exon 51 skip, No result
0
   0.0%
2
   1.7%
DMD Exon 51 skip, Not analyzed
2
   3.4%
2
   1.7%
13.Secondary Outcome
Title Change From Baseline in Pediatric Quality of Life (PedsQL) Total Score at Week 48
Hide Description PedsQL version 3.0 scale is used to measure pediatric quality of life in children with neuromuscular disorders. The 25-item PedsQL encompasses 3 scales About My/My Childs Neuromuscular Disease (17 items), Communication (3 items), About Our Family Resources (5 items). A 5-point response scale is utilized (where 0=never a problem; 4=almost always a problem). It was assessed both by child and parent. PedsQL total score was calculated by reverse scoring individual items and linearly transforming the score to a 0-100 scale, where higher scores indicated better health-related quality of life. To reverse score individual items, the 0-4 scale items were transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, and 4=0. Total score was then calculated as sum of items divided by number of items answered. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48. A positive change from Baseline indicated improvement.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the specified time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Assesor child at week 48 Number Analyzed 57 participants 114 participants
0.52  (11.313) 1.36  (11.360)
Assesor parent at week 48 Number Analyzed 58 participants 117 participants
-0.11  (11.064) -1.19  (11.269)
14.Secondary Outcome
Title Change From Baseline in Pulmonary Function Test Peak Cough Flow (PCF) and Peak Flow (PF) at Week 48
Hide Description The PF also called peak expiratory flow rate (PEFR) is a participants maximum speed of expiration, as measured with a peak flow meter, a small, hand-held device used to monitor a participants ability to breathe out air. PCF was measured for participants wearing a nose clip and performing a maximum cough into a pocket peak flow meter. Baseline was defined as participants randomization assessment at Visit 3 (Day 0). Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Day 0) and Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants available at the specified time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Mean (Standard Deviation)
Unit of Measure: Liters per minute
PF at Week 48 Number Analyzed 59 participants 121 participants
25.810  (41.5342) 11.603  (42.6964)
PCF at Week 48 Number Analyzed 59 participants 120 participants
22.7  (42.46) 10.7  (45.04)
15.Secondary Outcome
Title Number of Participants Who Showed Improvement on Clinician Global Impression of Improvement (CGI-I) Scale at Week 48
Hide Description The CGI-I is scored based on the clinician’s reflection of the participant’s current overall clinical condition compared to the overall clinical condition just prior to the initiation of medication use (i.e., the period prior to Randomization). The CGI-I is rated without regard to the clinician’s belief that any clinical changes are or are not due to medication and without consideration of the etiology of the symptoms. The CGI-I is measured on a 7-point Likert scale (where 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse). The score ranged from 1-7, where lower score indicated more improvement and higher score indicated less improvement.
Time Frame Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Only those participants with data available at the indicated time point were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 57 116
Measure Type: Count of Participants
Unit of Measure: Participants
Very much improved
0
   0.0%
3
   2.6%
Much improved
1
   1.8%
9
   7.8%
Minimally improved
2
   3.5%
23
  19.8%
No change
27
  47.4%
41
  35.3%
Minimally worse
20
  35.1%
30
  25.9%
Much worse
6
  10.5%
10
   8.6%
Very much worse
1
   1.8%
0
   0.0%
16.Secondary Outcome
Title Change From Baseline in Health Utilities Index (HUI) Scores at Week 48
Hide Description A 15-item HUI questionnaire assessed Health-related quality of life (HRQoL). Responses from 15-item HUI were used to quantify HRQoL according to 2 health status classification systems, HUI Mark 2 (HUI2) and HUI Mark 3 (HUI3). HUI2 assessed 7 HRQoL dimensions: sensation, mobility, emotion, cognition, self care, pain and fertility. HUI3 assessed 8 HRQoL dimensions: vision, hearing, speech, ambulation, dexterity, emotion, cognition and pain. Both HUI2 (range from -0.03 to 1.0) and HUI3 (range from -0.36 to 1.0) utility scores were calculated using algorithms incorporating community-derived preference weights. A utility value of 1.0 represented perfect health and a utility value of 0.0 represented death. Lowest possible HUI2 score was -0.03 and for HUI3 score was -0.36, where scores less than 0 represented health states considered worse than death. Change from Baseline was calculated by subtracting Baseline value from Week 48 value. A positive change from Baseline indicated improvement.
Time Frame Baseline (Randomization Visit, Day 0) and Week 48
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ITT Population. Only those participants with data available at the specified time points were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Least Squares Mean (Standard Error)
Unit of Measure: Scores on a scale
HUI2 at Week 48 Number Analyzed 53 participants 112 participants
-0.052  (0.0190) -0.023  (0.0133)
HUI3 at Week 48 Number Analyzed 56 participants 116 participants
-0.061  (0.0266) -0.056  (0.0188)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo, HUI2 at Week 48
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.207
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 for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.0288
Confidence Interval (2-Sided) 95%
-0.0161 to 0.0738
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, GSK2402968 6mg/kg/Week
Comments GSK2402968 6mg/kg/week Vs Placebo, HUI3 at Week 48
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.880
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 for Repeated Measures
Comments Model included terms for treatment, visit, treatment by visit, country grouping, Baseline and Baseline by visit.
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.0048
Confidence Interval (2-Sided) 95%
-0.0580 to 0.0676
Estimation Comments [Not Specified]
17.Secondary Outcome
Title Number of Participants With Adverse Events (AE) and Severe Adverse Events (SAE)
Hide Description An AE is any untoward medical occurrence in a clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect.
Time Frame Up to Follow-up (Week 68)
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The Safety Population comprised of all participants who received at least one dose of study medication.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
Any AE
58
  95.1%
123
  98.4%
Any SAE
5
   8.2%
13
  10.4%
18.Secondary Outcome
Title Number of Participants With Vital Sign Data for Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) and Heart Rate (HR) of Potential Clinical Concern (PCC) at Any Visit Post-Baseline
Hide Description Blood pressure SBP, DBP and HR were recorded after five minutes of rest in a semi-supine position. The following changes from Baseline (Day 0) in vital signs were considered to be of potential clinical concern: DBP was defined as high (increase from Baseline >=20 and >=40 millimeters of mercury [mmHg] and low (decrease from Baseline >=20 and >=40 mmHg), SBP high (increase from Baseline >=10 and >=20 mmHg and low (decrease from Baseline >=10 and >=20 mmHg) and for HR high (increase from Baseline >=20 and >=40 beats per minute [bpm] and low (decrease from Baseline >=20 and >=40 bpm). Only those parameters for which a value of PCC was reported at any visit post-Baseline is presented.
Time Frame Up to Week 48
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Hide Analysis Population Description
Safety Population.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
DBP, High at any visit post-Baseline
22
  36.1%
52
  41.6%
HR, low at any visit post-Baseline
19
  31.1%
49
  39.2%
HR, high at any visit post-Baseline
18
  29.5%
32
  25.6%
SBP, low at any visit post-Baseline
8
  13.1%
7
   5.6%
SBP, high at any visit post-Baseline
28
  45.9%
72
  57.6%
19.Secondary Outcome
Title Number of Participants With Abnormal-clinically Significant Electrocardiogram (ECG) Findings at Any Visit Post-Baseline
Hide Description ECG measurements were carried out and the clinical interpretation of the ECG by the investigator was recorded as normal, abnormal but not clinically significant and abnormal clinically significant. The PCC ranges include, QT interval corrected for heart rate by Bazett’s formula (QTcB) or QT interval corrected for heart rate by Fridericia’s formula (QTcF) >450 milliseconds and any increase from Baseline of QTcB or QTcF. Participants were categorized as abnormal clinically significant based on the investigator’s judgment and PCC ranges. Data has been presented for number of participants with abnormal clinically significant findings at any visit post-Baseline.
Time Frame Up to Week 48
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Safety Population.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
5
   4.0%
20.Secondary Outcome
Title Number of Participants With Hematology Parameters of PCC at Any Visit Post-Baseline
Hide Description Laboratory samples were collected for analysis of hematology parameters. The PCC values for hematology parameters: hematocrit was 1.02 x Upper limit of normal (ULN), for hemoglobin was 1.03 x ULN, for lymphocytes was 0.81 x lower limit of normal (LLN), for platelet count was 0.67 x LLN and 1.57 x ULN, for total neutrophils was 0.83 x LLN, and that for white blood cell count was 0.67 x LLN and value of 1.82 x ULN. Only those parameters for which a value of PCC was reported at any visit post-Baseline have been presented.
Time Frame Up to Week 48
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Safety Population.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
Hematocrit > reference range high
12
  19.7%
27
  21.6%
Hemoglobin > reference range high
2
   3.3%
6
   4.8%
Lymphocytes < reference range low
6
   9.8%
9
   7.2%
Platelet count > reference range high
1
   1.6%
0
   0.0%
Platelet count < reference range low
0
   0.0%
1
   0.8%
Total neutrophils < reference range low
8
  13.1%
9
   7.2%
White blood cell count < reference range low
3
   4.9%
4
   3.2%
21.Secondary Outcome
Title Number of Participants With Coagulation Parameters of PCC at Any Visit Post-Baseline
Hide Description Laboratory samples were collected for analysis of coagulation parameters. The PCC values for coagulation parameters activated partial thromboplastin time (aPTT) was 1.5 x ULN and aPTT ratio also known as international normalized ration (INR) was 1.2 x ULN. Only those parameters for which a value of PCC was reported at any visit post-Baseline is presented.
Time Frame Up to Week 48
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Safety Population.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
aPTT > reference range high
6
   9.8%
6
   4.8%
INR > reference range high
11
  18.0%
32
  25.6%
22.Secondary Outcome
Title Number of Participants With Clinical Chemistry Parameters of PCC at Any Visit Post-Baseline
Hide Description Laboratory samples were collected for analysis of chemistry parameters. The PCC values for chemistry parameters for alanine amino transferase (ALT) plus total bilirubin (TB) was >=1.5 x ULN for TB and >=2 x ULN for ALT, for albumin was 0.86 x LLN, for asparatate amino transferase (AST) was >=2 x ULN, for calcium was 0.91 x LLN and 1.06 x ULN, for glucose was 0.71 x LLN and 1.41 x ULN, for phosphorus was 0.80 x LLN and 1.14 x ULN, for sodium was 0.96 x LLN and 1.03 x ULN, for potassium was 0.86 x LLN and 1.10 x ULN and that for alkaline phosphatase was >=2x ULN. Only those parameters for which a value of PCC was reported at any visit post-Baseline is presented.
Time Frame Up to Week 48
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Safety Population.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
ALT plus TB > reference range high
0
   0.0%
2
   1.6%
ALT > reference range high
61
 100.0%
125
 100.0%
Albumin < reference range low
0
   0.0%
1
   0.8%
AST > reference range high
61
 100.0%
125
 100.0%
Calcium > reference range high
1
   1.6%
3
   2.4%
Calcium < reference range low
0
   0.0%
3
   2.4%
Glucose > reference range high
3
   4.9%
5
   4.0%
Glucose < reference range low
0
   0.0%
2
   1.6%
Phosphorus > reference range high
1
   1.6%
8
   6.4%
Sodium > reference range high
2
   3.3%
9
   7.2%
Potassium > reference range high
0
   0.0%
0
   0.0%
Potassium < reference range low
0
   0.0%
0
   0.0%
Alkaline phosphatase > reference range high
0
   0.0%
0
   0.0%
23.Secondary Outcome
Title Number of Participants With Urinalysis Data Outside the Reference Range (>Reference Range High) at Any Visit Post- Baseline
Hide Description Urine samples were collected for analysis of abnormal urine parameters. Quantitative examination included the assessment for urine albumin excretion rate, urine alpha-1-microglobulin, urine creatinine excretion-24 hour and urine protein excretion-24 hour. Only those parameters for which a value of >reference range high was reported at any visit post-Baseline is presented.
Time Frame Up to Week 48
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Safety Population. Only those participants available at the specified time points were analyzed.
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period.
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 61 125
Measure Type: Count of Participants
Unit of Measure: Participants
Urine Albumin excretion rate > reference range Number Analyzed 12 participants 93 participants
1
   8.3%
15
  16.1%
Urine Protein excretion -24 hour > reference range Number Analyzed 12 participants 96 participants
4
  33.3%
54
  56.3%
24.Secondary Outcome
Title Plasma Concentrations of GSK2402968 Following Subcutaneous Administration
Hide Description Blood samples for pharmacokinetic assessment were taken at Week 0 (Randomization) at 0.5, 1, 3 hours post-dose and at Week 8,12, 24, 36 and 47 at pre-dose, and between 1 and 4 hours post-dose. Data has been presented for plasma concentrations of GSK2402968 following subcutaneous administration.
Time Frame Randomization (Week 0 at 0.5, 1 and 3 hours), Week 8 (pre-dose, 1-4 hours), Week 12 (pre-dose, 1-4 hours), Week 24 (pre-dose, 1-4 hours), Week 36 (pre-dose, 1-4 hours), Week 47 (pre-dose, 1-4 hours)
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Hide Analysis Population Description
The Pharmacokinetic Population comprised all participants who were randomized to the study and from whom at least one blood sample was obtained for assessment of GSK2402968 concentration. Only those participants available at the specified time points were analyzed.
Arm/Group Title GSK2402968 6mg/kg/Week
Hide Arm/Group Description:
Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
Overall Number of Participants Analyzed 125
Median (Full Range)
Unit of Measure: Nanograms per millimeter
Week 0, 0.5 hours Number Analyzed 120 participants
3349.595
(572.07 to 10408.94)
Week 0, 1 hour Number Analyzed 122 participants
4946.950
(1094.90 to 13159.51)
Week 0, 3 hour Number Analyzed 121 participants
5932.740
(1794.00 to 12765.26)
Week 8, Pre-dose Number Analyzed 47 participants
12.730
(0.00 to 4424.28)
Week 8, 1-4 hours Number Analyzed 47 participants
5100.220
(7.62 to 10471.64)
Week 12, Pre-dose Number Analyzed 47 participants
19.090
(4.60 to 50.95)
Week 12, 1-4 hours Number Analyzed 47 participants
5613.040
(10.89 to 10583.33)
Week 24, Pre-dose Number Analyzed 49 participants
40.920
(0.00 to 186.87)
Week 24, 1-4 hours Number Analyzed 49 participants
6141.580
(40.65 to 11902.15)
Week 36, Pre-dose Number Analyzed 38 participants
55.185
(22.64 to 6590.81)
Week 36, 1-4 hours Number Analyzed 37 participants
5755.950
(68.84 to 12967.56)
Week 47, Pre-dose Number Analyzed 106 participants
61.395
(3.86 to 5922.31)
Week 47, 1-4 hours Number Analyzed 99 participants
5266.240
(46.42 to 13841.11)
Time Frame Up to Follow-up (Week 68).
Adverse Event Reporting Description The Safety Population was used for analysis.
 
Arm/Group Title Placebo GSK2402968 6mg/kg/Week
Hide Arm/Group Description Participants received single dose of matching placebo sterile solution for subcutaneous injection preferably in the morning over the 48 week treatment period. Participants received single dose of GSK2402968 6 mg/kg/week subcutaneous injection preferably in the morning over the 48 week treatment period.
All-Cause Mortality
Placebo GSK2402968 6mg/kg/Week
Affected / at Risk (%) Affected / at Risk (%)
Total   0/61 (0.00%)   0/125 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo GSK2402968 6mg/kg/Week
Affected / at Risk (%) Affected / at Risk (%)
Total   5/61 (8.20%)   13/125 (10.40%) 
Cardiac disorders     
Cardiac fibrillation  2  0/61 (0.00%)  1/125 (0.80%) 
Myocardial ischaemia  2  0/61 (0.00%)  1/125 (0.80%) 
Gastrointestinal disorders     
Enteritis  2  0/61 (0.00%)  1/125 (0.80%) 
General disorders     
Spinal pain  2  0/61 (0.00%)  1/125 (0.80%) 
Infections and infestations     
Gastroenteritis  2  1/61 (1.64%)  0/125 (0.00%) 
Viral infection  2  1/61 (1.64%)  0/125 (0.00%) 
Injury, poisoning and procedural complications     
Fall  1  0/61 (0.00%)  1/125 (0.80%) 
Femur fracture  2  0/61 (0.00%)  2/125 (1.60%) 
Head injury  2  0/61 (0.00%)  1/125 (0.80%) 
Lumbar vertebral fracture  2  0/61 (0.00%)  1/125 (0.80%) 
Patella fracture  2  1/61 (1.64%)  0/125 (0.00%) 
Toxicity to various agents  2  1/61 (1.64%)  0/125 (0.00%) 
Wound dehiscence  2  0/61 (0.00%)  1/125 (0.80%) 
Tibia fracture  2  0/61 (0.00%)  1/125 (0.80%) 
Investigations     
Alanine aminotransferase increased  2  0/61 (0.00%)  1/125 (0.80%) 
International normalised ratio increased  2  0/61 (0.00%)  1/125 (0.80%) 
Metabolism and nutrition disorders     
Dehydration  2  0/61 (0.00%)  1/125 (0.80%) 
Nervous system disorders     
Benign intracranial hypertension  2  0/61 (0.00%)  1/125 (0.80%) 
Intercostal neuralgia  2  1/61 (1.64%)  0/125 (0.00%) 
Intracranial venous sinus thrombosis  2  0/61 (0.00%)  1/125 (0.80%) 
Renal and urinary disorders     
Glomerulonephritis  2  0/61 (0.00%)  1/125 (0.80%) 
1
Term from vocabulary, MedDRA
2
Term from vocabulary, MedDRA 16.0
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 GSK2402968 6mg/kg/Week
Affected / at Risk (%) Affected / at Risk (%)
Total   54/61 (88.52%)   119/125 (95.20%) 
Ear and labyrinth disorders     
Ear pain  1  4/61 (6.56%)  6/125 (4.80%) 
Gastrointestinal disorders     
Vomiting  1  13/61 (21.31%)  28/125 (22.40%) 
Diarrhoea  1  9/61 (14.75%)  23/125 (18.40%) 
Abdominal pain  1  7/61 (11.48%)  14/125 (11.20%) 
Abdominal pain upper  1  3/61 (4.92%)  9/125 (7.20%) 
General disorders     
Injection site erythema  1  4/61 (6.56%)  62/125 (49.60%) 
Injection site discolouration  1  2/61 (3.28%)  41/125 (32.80%) 
Pyrexia  1  15/61 (24.59%)  27/125 (21.60%) 
Injection site pain  1  2/61 (3.28%)  23/125 (18.40%) 
Injection site reaction  1  1/61 (1.64%)  24/125 (19.20%) 
Injection site bruising  1  6/61 (9.84%)  11/125 (8.80%) 
Injection site induration  1  0/61 (0.00%)  17/125 (13.60%) 
Injection site pruritus  1  0/61 (0.00%)  17/125 (13.60%) 
Injection site haematoma  1  2/61 (3.28%)  9/125 (7.20%) 
Injection site atrophy  1  0/61 (0.00%)  9/125 (7.20%) 
Injection site urticaria  1  0/61 (0.00%)  9/125 (7.20%) 
Injection site swelling  1  0/61 (0.00%)  8/125 (6.40%) 
Infections and infestations     
Nasopharyngitis  1  25/61 (40.98%)  38/125 (30.40%) 
Upper respiratory tract infection  1  8/61 (13.11%)  15/125 (12.00%) 
Gastroenteritis  1  5/61 (8.20%)  15/125 (12.00%) 
Rhinitis  1  3/61 (4.92%)  10/125 (8.00%) 
Influenza  1  4/61 (6.56%)  7/125 (5.60%) 
Ear infection  1  5/61 (8.20%)  4/125 (3.20%) 
Pharyngitis  1  4/61 (6.56%)  5/125 (4.00%) 
Bronchitis  1  4/61 (6.56%)  4/125 (3.20%) 
Injury, poisoning and procedural complications     
Fall  1  12/61 (19.67%)  26/125 (20.80%) 
Contusion  1  7/61 (11.48%)  8/125 (6.40%) 
Excoriation  1  3/61 (4.92%)  7/125 (5.60%) 
Ligament sprain  1  3/61 (4.92%)  7/125 (5.60%) 
Investigations     
Protein urine present  1  4/61 (6.56%)  17/125 (13.60%) 
Red blood cells urine positive  1  4/61 (6.56%)  14/125 (11.20%) 
Cystatin C increased  1  2/61 (3.28%)  14/125 (11.20%) 
Red blood cells urine  1  4/61 (6.56%)  11/125 (8.80%) 
Urine protein/creatinine ratio increased  1  2/61 (3.28%)  11/125 (8.80%) 
Blood fibrinogen decreased  1  6/61 (9.84%)  3/125 (2.40%) 
Musculoskeletal and connective tissue disorders     
Pain in extremity  1  12/61 (19.67%)  11/125 (8.80%) 
Back pain  1  5/61 (8.20%)  8/125 (6.40%) 
Arthralgia  1  1/61 (1.64%)  10/125 (8.00%) 
Muscle spasms  1  4/61 (6.56%)  5/125 (4.00%) 
Nervous system disorders     
Headache  1  11/61 (18.03%)  33/125 (26.40%) 
Renal and urinary disorders     
Proteinuria  1  11/61 (18.03%)  42/125 (33.60%) 
Haematuria  1  5/61 (8.20%)  21/125 (16.80%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  12/61 (19.67%)  24/125 (19.20%) 
Epistaxis  1  5/61 (8.20%)  11/125 (8.80%) 
Oropharyngeal pain  1  1/61 (1.64%)  10/125 (8.00%) 
Skin and subcutaneous tissue disorders     
Rash  1  6/61 (9.84%)  3/125 (2.40%) 
Erythema  1  1/61 (1.64%)  7/125 (5.60%) 
Pruritus  1  1/61 (1.64%)  7/125 (5.60%) 
1
Term from vocabulary, MedDRA 16.0
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: NCT01254019     History of Changes
Other Study ID Numbers: 114044
First Submitted: October 21, 2010
First Posted: December 6, 2010
Results First Submitted: September 21, 2017
Results First Posted: January 28, 2019
Last Update Posted: January 28, 2019