Trial · NCT01254019

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

NCT01254019, Phase 3, Completed, GlaxoSmithKline; results first posted 2019-01-28

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy. Trials, record NCT01254019. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01254019

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
NCT number: NCT01254019
Status: Completed
Phases: Phase 3
Enrollment: 186 participants, actual
Lead sponsor: GlaxoSmithKline
Conditions: Muscular Dystrophies
Interventions: GSK2402968 6mg/kg/week
First posted: 2010-12-06
Last updated: 2019-01-28
Results first posted: 2019-01-28

Source ClinicalTrials.gov

Registered outcomes24

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Change From Baseline in the Linearized North Star Ambulatory Assessment (NSAA) Total Score at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Change From Baseline in the 4 Stair Climb (Ascent) Velocity at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Change From Baseline in the 10-meter Walk/Run Velocity at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Change From Baseline in the Timed Function Test Rise From Floor at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Change From Baseline in the 4 Stair Climb (Descent) Velocity at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Change From Baseline in Muscle Strength (Total Score) at Week 48	Baseline (Day 0) and Week 48	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.
Secondary	Kaplan-Meier Estimates for Time to Loss of Ambulation	Week 48	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.
Secondary	Number of Participants Who Experienced Accidental Falls During 6MWD Assessments at Week 48	Week 48	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.
Secondary	Change From Baseline in Creatine Kinase Serum Concentrations at Week 48	Baseline (Day 0) and Week 48	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.

1 to 10 of 24Source ClinicalTrials.gov

See all 24 registered outcomes

Outcomes24

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48	Baseline (Day 0) and Week 48	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.	Meters	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the Linearized North Star Ambulatory Assessment (NSAA) Total Score at Week 48	Baseline (Day 0) and Week 48	ITT Population. Only those participants available at the indicated time point were analyzed.	Scores on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the 4 Stair Climb (Ascent) Velocity at Week 48	Baseline (Day 0) and Week 48	ITT population. Only those participants available at the indicated time point were analyzed.	Stairs per second	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the 10-meter Walk/Run Velocity at Week 48	Baseline (Day 0) and Week 48	ITT Population. Only those participants available at the indicated time point were analyzed.	Meters per second	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the Timed Function Test Rise From Floor at Week 48	Baseline (Day 0) and Week 48	ITT Population. Only those participants available at the indicated time point were analyzed.	Seconds	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in the 4 Stair Climb (Descent) Velocity at Week 48	Baseline (Day 0) and Week 48	ITT Population. Only those participants available at the indicated time point were analyzed.	Stairs per second	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline in Muscle Strength (Total Score) at Week 48	Baseline (Day 0) and Week 48	ITT Population. Only those participants available at the indicated time point were analyzed.	Pounds	LEAST_SQUARES_MEAN
SECONDARY	Kaplan-Meier Estimates for Time to Loss of Ambulation	Week 48	ITT Population.	Days	MEDIAN
SECONDARY	Number of Participants Who Experienced Accidental Falls During 6MWD Assessments at Week 48	Week 48	ITT Population. Only those participants with data available at the indicated time point were analyzed.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Change From Baseline in Creatine Kinase Serum Concentrations at Week 48	Baseline (Day 0) and Week 48	ITT Population. Only those participants available at the indicated time point were analyzed.	International units per liter	LEAST_SQUARES_MEAN

1 to 10 of 24Source ClinicalTrials.gov

See all 24 outcomes

Results by group137

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48	Placebo	-52.65	Standard Error 10.423	Meters
Change From Baseline in Muscle Function Using the 6 Minute Walking Distance (6MWD) Test Assessed at Week 48	GSK2402968 6mg/kg/Week	-42.32	Standard Error 7.378	Meters
Change From Baseline in the Linearized North Star Ambulatory Assessment (NSAA) Total Score at Week 48	Placebo	-6.7	Standard Error 1.43	Scores on a scale
Change From Baseline in the Linearized North Star Ambulatory Assessment (NSAA) Total Score at Week 48	GSK2402968 6mg/kg/Week	-7.2	Standard Error 1.01	Scores on a scale
Change From Baseline in the 4 Stair Climb (Ascent) Velocity at Week 48	Placebo	-0.12	Standard Error 0.049	Stairs per second
Change From Baseline in the 4 Stair Climb (Ascent) Velocity at Week 48	GSK2402968 6mg/kg/Week	-0.14	Standard Error 0.035	Stairs per second
Change From Baseline in the 10-meter Walk/Run Velocity at Week 48	Placebo	-0.20	Standard Error 0.050	Meters per second
Change From Baseline in the 10-meter Walk/Run Velocity at Week 48	GSK2402968 6mg/kg/Week	-0.21	Standard Error 0.035	Meters per second
Change From Baseline in the Timed Function Test Rise From Floor at Week 48	Placebo	7.48	Standard Error 2.080	Seconds
Change From Baseline in the Timed Function Test Rise From Floor at Week 48	GSK2402968 6mg/kg/Week	6.36	Standard Error 1.463	Seconds

1 to 10 of 137Source ClinicalTrials.gov

See all 137 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo	61
Overall Study	STARTED	GSK2402968 6mg/kg/Week	125
Overall Study	COMPLETED	Placebo	60
Overall Study	COMPLETED	GSK2402968 6mg/kg/Week	121
Overall Study	NOT COMPLETED	Placebo	1
Overall Study	NOT COMPLETED	GSK2402968 6mg/kg/Week	4

Source ClinicalTrials.gov

Adverse events138

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Injection site erythema	General disorders	Placebo	4	61
other	Injection site erythema	General disorders	GSK2402968 6mg/kg/Week	62	125
other	Injection site discolouration	General disorders	Placebo	2	61
other	Injection site discolouration	General disorders	GSK2402968 6mg/kg/Week	41	125
other	Pyrexia	General disorders	Placebo	15	61
other	Pyrexia	General disorders	GSK2402968 6mg/kg/Week	27	125
other	Injection site pain	General disorders	Placebo	2	61
other	Injection site pain	General disorders	GSK2402968 6mg/kg/Week	23	125
other	Injection site reaction	General disorders	Placebo	1	61
other	Injection site reaction	General disorders	GSK2402968 6mg/kg/Week	24	125

1 to 10 of 138Source ClinicalTrials.gov

See all 138 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo	0	61
Total, all-cause mortality	GSK2402968 6mg/kg/Week	0	125
Total, other adverse events	Placebo	54	61
Total, other adverse events	GSK2402968 6mg/kg/Week	119	125
Total, serious adverse events	Placebo	5	61
Total, serious adverse events	GSK2402968 6mg/kg/Week	13	125

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
29203355	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
GSK2402968 6mg/kg/Week vs Placebo: Number of Participants With Abnormal-clinically Significant Electrocardiogram (ECG) Findings at Any Visit Post-Baseline	Trial comparisons
GSK2402968 6mg/kg/Week vs Placebo: deaths from any cause	Trial comparisons
GSK2402968 6mg/kg/Week vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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