Trial Outcomes & Findings for A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) (NCT NCT01462292)

Last Updated: 2017-10-16

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

51 participants

Primary outcome timeframe

Baseline (Week 0) and Week 24

Results posted on

2017-10-16

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Placebo (Combined) 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.	GSK2402968 3 mg/kg/Week 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	GSK2402968 6 mg/kg/Week 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 Study STARTED	16	17	18
Overall Study COMPLETED	16	17	18
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo (Combined) n=16 Participants 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.	GSK2402968 3 mg/kg/Week n=17 Participants 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.	GSK2402968 6 mg/kg/Week n=18 Participants 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 n=51 Participants Total of all reporting groups
Age, Continuous	8.0 years STANDARD_DEVIATION 1.79 • n=93 Participants	7.8 years STANDARD_DEVIATION 1.91 • n=4 Participants	7.6 years STANDARD_DEVIATION 2.70 • n=27 Participants	7.8 years STANDARD_DEVIATION 2.15 • n=483 Participants
Sex: Female, Male Female	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Sex: Female, Male Male	16 Participants n=93 Participants	17 Participants n=4 Participants	18 Participants n=27 Participants	51 Participants n=483 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	2 Participants n=27 Participants	2 Participants n=483 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants	3 Participants n=483 Participants
Race (NIH/OMB) White	14 Participants n=93 Participants	16 Participants n=4 Participants	15 Participants n=27 Participants	45 Participants n=483 Participants
Race (NIH/OMB) More than one race	1 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	1 Participants n=483 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants

PRIMARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: 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

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Mean Change From Baseline in Muscle Function Using the 6 Minute Walking Distance	-10.98 Meters Standard Error 10.666	-19.93 Meters Standard Error 9.964	16.12 Meters Standard Error 9.941

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT Population

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Change From Baseline in Rise From Floor Time at Week 24	1.12 Seconds Standard Error 0.700	1.50 Seconds Standard Error 0.662	1.95 Seconds Standard Error 0.656

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT Population

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Change From Baseline in 4 Stair Climb Ascent/Descent Time at Week 24 Ascent time	0.59 seconds Standard Error 0.308	0.59 seconds Standard Error 0.295	-0.22 seconds Standard Error 0.295
Change From Baseline in 4 Stair Climb Ascent/Descent Time at Week 24 Descent time	0.60 seconds Standard Error 0.473	-0.09 seconds Standard Error 0.442	0.19 seconds Standard Error 0.440

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT population.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Change From Baseline in 10 Meter Walk/Run at Week 24	-0.04 Seconds Standard Error 0.196	0.52 Seconds Standard Error 0.187	-0.01 Seconds Standard Error 0.185

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT Population. Only those participants available at the specified timepoints were analyzed

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=17 Participants 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.
Change From Baseline in Muscle Strength Total Score at Week 24	0.32 Pounds (lbs) Standard Error 4.633	2.67 Pounds (lbs) Standard Error 4.385	1.14 Pounds (lbs) Standard Error 4.469

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT population.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Shoulder abductor right	0.29 lbs Standard Deviation 1.588	0.26 lbs Standard Deviation 2.150	0.69 lbs Standard Deviation 2.106
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Knee extensor left	0.01 lbs Standard Deviation 4.574	-1.14 lbs Standard Deviation 3.219	1.02 lbs Standard Deviation 3.863
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Knee extensor right	-0.99 lbs Standard Deviation 3.625	-0.88 lbs Standard Deviation 3.123	0.45 lbs Standard Deviation 3.235
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Knee flexor left	0.10 lbs Standard Deviation 3.398	0.37 lbs Standard Deviation 2.178	0.48 lbs Standard Deviation 2.211
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Knee flexor right	0.11 lbs Standard Deviation 2.763	0.05 lbs Standard Deviation 2.410	0.28 lbs Standard Deviation 2.039
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Hip flexor left	-0.53 lbs Standard Deviation 2.922	-0.58 lbs Standard Deviation 2.848	1.10 lbs Standard Deviation 3.391
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Hip flexor right	-0.82 lbs Standard Deviation 4.190	-0.08 lbs Standard Deviation 3.213	0.57 lbs Standard Deviation 3.243
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Elbow flexor left	0.86 lbs Standard Deviation 2.443	0.56 lbs Standard Deviation 2.410	0.69 lbs Standard Deviation 2.273
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Elbow flexor right	0.47 lbs Standard Deviation 2.633	-0.25 lbs Standard Deviation 1.737	0.11 lbs Standard Deviation 1.801
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Elbow extensor left	0.50 lbs Standard Deviation 1.344	0.45 lbs Standard Deviation 2.235	0.56 lbs Standard Deviation 2.547
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Elbow extensor right	0.69 lbs Standard Deviation 1.673	1.11 lbs Standard Deviation 2.118	0.15 lbs Standard Deviation 1.715
Change From Baseline in Muscle Strength Tests For-Knee Extensor, Knee Flexor, Hip Flexor, Elbow Flexor, Elbow Extensor, Shoulder Abductor at Week 24 Shoulder abductor left	0.13 lbs Standard Deviation 1.423	0.65 lbs Standard Deviation 1.825	0.31 lbs Standard Deviation 1.710

SECONDARY outcome

Timeframe: Baselie (Week 0) and Week 24

Population: ITT Population

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score	-0.6 Scores on scale Standard Error 0.84	-1.1 Scores on scale Standard Error 0.80	-0.8 Scores on scale Standard Error 0.79

SECONDARY outcome

Timeframe: Baseline (Week 0), Week 24, Week 36 and Week 48

Population: ITT Population. Only those participants available at the specified timepoints were analyzed

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 12, 0 fall	15 Participants	17 Participants	17 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 12, 1 fall	1 Participants	0 Participants	1 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 24, 0 fall	16 Participants	16 Participants	16 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 24, 1 fall	0 Participants	1 Participants	2 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 36, 0 fall	14 Participants	13 Participants	14 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 36, 2 falls	0 Participants	1 Participants	0 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 48, 0 fall	10 Participants	14 Participants	13 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Week 48, 3 falls	1 Participants	0 Participants	0 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Baseline, 0 fall	16 Participants	16 Participants	17 Participants
Number of Participants With Accidental Falls During 6 Minute Walk Distance Test Baseline, 1 fall	0 Participants	1 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 48

Population: ITT population. Only those participants available at the specified time points were analyzed.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Change From Baseline in Creatinine Kinase Serum Concentrations Week 24	-3303.5 International units per liter Standard Error 1224.65	-4196.4 International units per liter Standard Error 1171.20	-4609.0 International units per liter Standard Error 1170.75
Change From Baseline in Creatinine Kinase Serum Concentrations Week 48	-2783.1 International units per liter Standard Error 1248.65	-4838.0 International units per liter Standard Error 1170.75	-2615.7 International units per liter Standard Error 1149.44

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT Population. Only those participants available at the indicated timepoints were used for analysis

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=16 Participants 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	GSK2402968 6 mg/kg/Week n=15 Participants 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.
Change From Baseline in Forced Expiratory Volume in the First Second of Exhalation (FEV1) at Week 24	0.054 Litres Standard Deviation 0.2445	-0.009 Litres Standard Deviation 0.2316	0.015 Litres Standard Deviation 0.1647

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT population. Only those participants available at the indicated timepoints were used for analysis.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=16 Participants 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	GSK2402968 6 mg/kg/Week n=15 Participants 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.
Change From Baseline in Forced Vital Capacity (FVC) at Week 24	0.063 Litres Standard Deviation 0.2670	0.080 Litres Standard Deviation 0.2064	0.051 Litres Standard Deviation 0.1419

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT population. Only those participants available at the indicated timepoints were used for analysis

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=15 Participants 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	GSK2402968 3 mg/kg/Week n=15 Participants 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	GSK2402968 6 mg/kg/Week n=17 Participants 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.
Change From Baseline in Peak Cough Flow at Week 24	16.5 Litres per minute Standard Deviation 19.03	18.2 Litres per minute Standard Deviation 24.19	18.0 Litres per minute Standard Deviation 28.09

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT population. Only those participants available at the specified time points were analyzed

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=16 Participants 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	GSK2402968 6 mg/kg/Week n=17 Participants 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.
Change From Baseline in Peak Expiratory Flow at Week 24	18.9 Litres per minute Standard Deviation 35.89	2.5 Litres per minute Standard Deviation 35.98	6.6 Litres per minute Standard Deviation 34.68

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT Population. Only those participants available at the specified time points were analyzed

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=15 Participants 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	GSK2402968 6 mg/kg/Week n=16 Participants 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.
Change From Baseline in Sniff Pressure Test at Week 24	3.46 Centimeter of water Standard Deviation 12.988	-1.47 Centimeter of water Standard Deviation 10.155	2.75 Centimeter of water Standard Deviation 16.838

SECONDARY outcome

Timeframe: Baseline (Week 0) and Week 24

Population: ITT population. Only those participants available at the specified timepoints were analyzed.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=12 Participants 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	GSK2402968 3 mg/kg/Week n=11 Participants 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	GSK2402968 6 mg/kg/Week n=13 Participants 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.
Number of Participants With Change From Baseline in Dystrophin Expression at Week 24 by Immunofluorescence Assay (IFA) Strong increase	5 Participants	3 Participants	0 Participants
Number of Participants With Change From Baseline in Dystrophin Expression at Week 24 by Immunofluorescence Assay (IFA) Increase	2 Participants	2 Participants	1 Participants
Number of Participants With Change From Baseline in Dystrophin Expression at Week 24 by Immunofluorescence Assay (IFA) Decrease	2 Participants	3 Participants	4 Participants
Number of Participants With Change From Baseline in Dystrophin Expression at Week 24 by Immunofluorescence Assay (IFA) No change	3 Participants	3 Participants	8 Participants

SECONDARY outcome

Timeframe: Week 24 and Week 48

Population: ITT population. Only those participants available at the specified time points were analyzed.

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').

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Number of Clinician Global Impression of Improvement (CGI-I) Responders Week 24	2 Participants	1 Participants	2 Participants
Number of Clinician Global Impression of Improvement (CGI-I) Responders Week 48	1 Participants	0 Participants	2 Participants

SECONDARY outcome

Timeframe: Up to Week 24

Population: ITT population. Only those participants available at that particular time points were analyzed.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to climb 3-4 steps, not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to climb 10-15 steps, improved	2 Participants	2 Participants	3 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to climb 10-15 steps, not improved	14 Participants	14 Participants	14 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk up/down a slope, not improved	11 Participants	12 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk up/down a slope, not applicable	1 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to run and kick a ball, improved	4 Participants	1 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to get in and out of bed, improved	4 Participants	4 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to get in and out of car, improved	4 Participants	3 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to get in and out of car, not applicable	0 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to play with toys, not applicable	1 Participants	0 Participants	2 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to zip jacket or pants, not applicable	1 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Childs need for orthotics, not improved	9 Participants	10 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to brush teeth, not improved	14 Participants	15 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Ability to walk 50 feet, improved	4 Participants	4 Participants	8 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Ability to walk 50 feet, not improved	12 Participants	13 Participants	10 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Ability to walk 50 feet, not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Rate your child's condition, improved	5 Participants	6 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Rate your child's condition, not improved	11 Participants	11 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Walk 15-20 mins without tired,improved	5 Participants	5 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Walk 15-20 mins without tired,not improved	11 Participants	11 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Walk 15-20 mins without tired,not applicable	0 Participants	1 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to stand 10 mins without tired, improved	3 Participants	4 Participants	4 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to stand 10 mins without tired,not improved	13 Participants	13 Participants	14 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to stand 10 mins without tired,not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to step up on stool, improved	3 Participants	5 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to step up on stool, not improved	13 Participants	11 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to step up on stool, not applicable	0 Participants	1 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk up/down a slope, improved	4 Participants	5 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to climb 3-4 steps, improved	2 Participants	5 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to climb 3-4 steps, not improved	14 Participants	12 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to climb 10-15 steps, not applicable	0 Participants	1 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk on uneven surfaces, improved	4 Participants	3 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk on uneven surfaces, not improved	12 Participants	14 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk on uneven surfaces, not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk without fall, improved	6 Participants	5 Participants	8 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk without fall, not improved	10 Participants	12 Participants	10 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to walk without fall, not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Participate in physical activities, improved	4 Participants	3 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Participate in physical activities, not improved	12 Participants	13 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Participate in physical activities, not applicable	0 Participants	1 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to go out with friends/family, improved	6 Participants	5 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to go out with friends/family, not improved	10 Participants	12 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to go out with friends/family, not applicable	0 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to throw and catch ball, improved	3 Participants	2 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to throw and catch ball, not improved	13 Participants	15 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to throw and catch ball, not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to run and kick a ball, not improved	12 Participants	15 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to run and kick a ball, not applicable	0 Participants	1 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to get in and out of bed, not improved	12 Participants	13 Participants	11 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to get in and out of bed, not applicable	0 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to get in and out of car, not improved	12 Participants	14 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to pick small item from table, improved	1 Participants	2 Participants	4 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to pick small item from table, not improved	15 Participants	15 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to pick small item from table, not applicable	0 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to pick large item from table, improved	2 Participants	3 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to pick large item from table, not improved	14 Participants	14 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to play with toys, improved	1 Participants	4 Participants	3 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to play with toys, not improved	14 Participants	13 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to play board or video games, improved	4 Participants	4 Participants	7 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to play board or video games, not improved	12 Participants	13 Participants	11 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to play board or video games, not applicable	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to work on computer, improved	3 Participants	2 Participants	4 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to work on computer, not improved	13 Participants	15 Participants	12 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to work on computer, not applicable	0 Participants	0 Participants	2 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to squeeze out toothpaste, improved	1 Participants	2 Participants	3 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to squeeze out toothpaste, not improved	15 Participants	12 Participants	14 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to squeeze out toothpaste, not applicable	0 Participants	3 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to brush teeth, improved	2 Participants	2 Participants	4 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to brush teeth, not applicable	0 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Pour milk or juice from in a cup , improved	2 Participants	0 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Pour milk or juice from in a cup, not improved	14 Participants	16 Participants	11 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Pour milk or juice from in a cup, not applicable	0 Participants	1 Participants	2 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to drink from cup without straw, improved	2 Participants	2 Participants	4 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to drink from cup without straw, not improved	14 Participants	15 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Drink from cup without straw, not applicable	0 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to button shirt or jacket, improved	2 Participants	0 Participants	6 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to button shirt or jacket, not improved	14 Participants	16 Participants	10 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to button shirt or jacket, not applicable	0 Participants	1 Participants	2 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to zip jacket or pants, improved	1 Participants	1 Participants	4 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Able to zip jacket or pants, not improved	14 Participants	16 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Childs need for orthotics, improved	0 Participants	0 Participants	0 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Childs need for orthotics, not applicable	7 Participants	7 Participants	13 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Childs need for wheelchair, improved	1 Participants	0 Participants	1 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Childs need for wheelchair, not improved	6 Participants	10 Participants	5 Participants
Assessment of Functional Outcome by : Functional Outcomes Survey During Treatment Period Childs need for wheelchair, not applicable	9 Participants	7 Participants	12 Participants

SECONDARY outcome

Timeframe: Week 24 and Week 48

Population: ITT population. Only those participants available at the specified time points were analyzed.

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.

Outcome measures

Outcome measures
Measure	Placebo (Combined) n=16 Participants 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	GSK2402968 3 mg/kg/Week n=17 Participants 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	GSK2402968 6 mg/kg/Week n=18 Participants 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.
Assessment of Functional Outcome by : Physician Assessment of Daily Living Week 12, Any improvement	9 Participants	6 Participants	12 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Week 12, Any worsening	2 Participants	1 Participants	0 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Week 24, Any improvement	10 Participants	8 Participants	13 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Week 24, Any worsening	5 Participants	3 Participants	3 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Week 48, Any improvement	5 Participants	5 Participants	7 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Week 48, Any worsening	6 Participants	6 Participants	3 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Anytime during study, Any improvement	12 Participants	11 Participants	17 Participants
Assessment of Functional Outcome by : Physician Assessment of Daily Living Anytime during study, Any worsening	11 Participants	8 Participants	6 Participants

Adverse Events

Placebo (Combined)

Serious events: 1 serious events

Other events: 13 other events

Deaths: 0 deaths

GSK2402968 3 mg/kg/Week

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

GSK2402968 6 mg/kg/Week

Serious events: 0 serious events

Other events: 17 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo (Combined) n=16 participants at risk 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	GSK2402968 3 mg/kg/Week n=17 participants at risk 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	GSK2402968 6 mg/kg/Week n=18 participants at risk 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
Infections and infestations Wound infection staphylococcal	6.2% 1/16 • Up to Week 48 Safety Population was defined as all participants who received at least one dose of study medication.	0.00% 0/17 • Up to Week 48 Safety Population was defined as all participants who received at least one dose of study medication.	0.00% 0/18 • Up to Week 48 Safety Population was defined as all participants who received at least one dose of study medication.

Other adverse events

Additional Information

GSK Response Center

GlaxoSmithKline

Phone: 866-435-7343

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER