Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01254019 | A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy | PHASE3 | COMPLETED | 186 | — | — | Dec 2, 2010 | Jun 28, 2013 | Jan 28, 2019 | 47 | Argentina, Belgium +18 |
| NCT01462292 | A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) | PHASE2 | COMPLETED | 51 | — | — | Oct 26, 2011 | Nov 4, 2013 | Oct 16, 2017 | 14 | United States |
| NCT01153932 | Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy | PHASE2 | COMPLETED | 53 | — | — | Sep 1, 2010 | Sep 1, 2012 | Aug 25, 2014 | 13 | Australia, Belgium +7 |
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.
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.
| Arm | Type | Description |
|---|---|---|
| GSK2402968 | EXPERIMENTAL | 6mg/kg |
| Placebo | EXPERIMENTAL | dose-matched |
| GSK2402968 3 mg/kg/week | EXPERIMENTAL | 3 mg/kg/week of investigational product |
| GSK2402968 6 mg/kg/week | EXPERIMENTAL | 6 mg/kg/week of investigational Product |
| Placebo to match GSK2402968 3 mg/kg/week | EXPERIMENTAL | Placebo |
| Placebo to match GSK2402968 6 mg/kg/week | EXPERIMENTAL | Placebo |
| Continuous regimen; 6mg/kg once weekly | EXPERIMENTAL | Once Weekly |
| Intermittent regimen; 6mg/kg twice weekly | EXPERIMENTAL | Twice 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 |
| Name | Type | Description |
|---|---|---|
| GSK2402968 6mg/kg/week | DRUG | subcutaneous |
| GSK2402968 3mg/kg/week | DRUG | Comparison of 2 doses of GSK2402968 |
| GSK2402968 6 mg/kg/week | DRUG | Comparison of 2 doses of GSK2402968 |
| Placebo to match GSK2402968 3 mg/kg/week | DRUG | Matched placebo |
| Placebo to match GSK2402968 6 mg/kg/week | DRUG | Matched Placebo |
| GSK2402968 | DRUG | Subcutaneous injection |
| matched placebo | DRUG | Subcutaneous injection |
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)...