drugset / Trial / NCT01462292
A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping. Two doses of GSK2402968 and placebo will be used in this study.
Timeline
- Start
- 2011-10-26
- Primary completion
- 2013-05-21
- Completion
- 2013-11-04
Outcome
Missed primary endpoint
registry analysis (superiority or other test); Placebo (Combined) vs GSK2402968 6 mg/kg/Week; p = 0.069; Mean Difference (Net) 27.099 (95% CI -2.210 to 56.408) NCT01462292 ↗
registry analysis (superiority or other test); Placebo (Combined) vs GSK2402968 3 mg/kg/Week; p = 0.554; Mean Difference (Net) -8.946 (95% CI -39.122 to 21.229) NCT01462292 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Drisapersen | Antisense oligonucleotide | 3 mg/kg | — |
| Subject | Drisapersen | Antisense oligonucleotide | 6 mg/kg | — |