welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

key information

study id #: NCT01128855

condition: Muscular Dystrophies

status: completed


The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy

3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968, 12 mg/kg GSK2402968, Placebo

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01128855

last updated: November 22, 2018