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GSK2402968/kg/week

Phase 3

Muscular Dystrophies | Small molecule | Neurology |GSK plc|Last Updated: Jan 28, 2019

Success Probability

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials3
Total Enrollment290

FDA Designations

No designations recorded

Clinical trial landscape

GSK2402968/kg/week · 3 trials · 1 indication

Phase 3 1Phase 2 2
NCT01254019A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular DystrophyMuscular Dystrophies
COMPLETED186 Analytics
PHASE3COMPLETED
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Muscular DystrophiesUnlock trial analytics

Study Endpoints

Primary Endpoints

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.

Mean Change From Baseline in Muscle Function Using the 6 Minute Walking Distance
Baseline (Week 0) and Week 24

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.

To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD
48 weeks

Secondary Endpoints

Change From Baseline in the Linearized North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Baseline (Day 0) and Week 48
Change From Baseline in the 4 Stair Climb (Ascent) Velocity at Week 48
Baseline (Day 0) and Week 48
Change From Baseline in the 10-meter Walk/Run Velocity at Week 48
Baseline (Day 0) and Week 48
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
GSK2402968EXPERIMENTAL6mg/kg
PlaceboEXPERIMENTALdose-matched
GSK2402968 3 mg/kg/weekEXPERIMENTAL3 mg/kg/week of investigational product
GSK2402968 6 mg/kg/weekEXPERIMENTAL6 mg/kg/week of investigational Product
Placebo to match GSK2402968 3 mg/kg/weekEXPERIMENTALPlacebo
Placebo to match GSK2402968 6 mg/kg/weekEXPERIMENTALPlacebo
Continuous regimen; 6mg/kg once weeklyEXPERIMENTALOnce Weekly
Intermittent regimen; 6mg/kg twice weeklyEXPERIMENTALTwice weekly on 1st, 3rd and 5th weeks, once weekly on 2nd, 4th and 6th weeks, and no active drug on 7th to 10th week of each 10 week cycle

Interventions

NameTypeDescription
GSK2402968 6mg/kg/weekDRUGsubcutaneous
GSK2402968 3mg/kg/weekDRUGComparison of 2 doses of GSK2402968
GSK2402968 6 mg/kg/weekDRUGComparison of 2 doses of GSK2402968
Placebo to match GSK2402968 3 mg/kg/weekDRUGMatched placebo
Placebo to match GSK2402968 6 mg/kg/weekDRUGMatched Placebo
GSK2402968DRUGSubcutaneous injection
matched placeboDRUGSubcutaneous injection
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Eligibility Criteria

Age Range5 Years to N/A
SexMALE
Healthy VolunteersNo
Study Sites47

Inclusion Criteria: * Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation/deletion within the DMD gene, confirmed by a state-of-the-art DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification)...

Countries:ArgentinaBelgiumBrazilCanadaChileCzechiaDenmarkFranceGermanyHungaryItalyJapanNetherlandsNorwayPolandRussiaSouth KoreaSpainTaiwanTurkey (Türkiye)United StatesAustraliaIsraelUnited Kingdom
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Frequently asked questions about GSK2402968/kg/week

What is GSK2402968 used for?

GSK2402968 is an investigational small molecule being developed for the treatment of muscular dystrophies, specifically Duchenne muscular dystrophy (DMD). It has been studied in clinical trials enrolling male subjects aged 5 years and older with this condition. The drug is not approved and remains in clinical development.

Who makes GSK2402968?

GSK2402968 is being developed by GSK plc, a global biopharmaceutical company traded on the London Stock Exchange under the ticker GSK. The company has sponsored clinical trials of this investigational drug in multiple countries across Europe, the Americas, Asia, and the Middle East.

What phase is GSK2402968 in?

GSK2402968 has completed Phase 2 and Phase 3 clinical trials. The most advanced study was a Phase 3 trial (NCT01254019) that has been completed. However, the drug is still considered investigational and has not been approved by regulatory authorities. No active trials are currently listed.

What clinical trials has GSK2402968 been in?

GSK2402968 has been studied in three completed clinical trials. NCT01153932 was a Phase 2 double-blind exploratory study in 53 ambulant subjects. NCT01254019 was a Phase 3 efficacy and safety study in 186 subjects. NCT01462292 was a Phase 2 dose-comparison study in 51 subjects in the United States.

Is GSK2402968 the same as other Duchenne muscular dystrophy drugs?

GSK2402968 is a distinct investigational small molecule developed specifically for Duchenne muscular dystrophy. It is not known to be the same as any other approved or investigational drug. Its mechanism of action has not been disclosed in the available clinical trial information.