DMD114117

• Research type

  Research Study

• Full title

  A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy

• IRAS ID

  48665

• Contact name

  Volker Straub

• Sponsor organisation

  GlaxoSmithKline Research and Development LTD

• Eudract number

  2010-018412-32

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  This is a Phase II study designed to assess efficacy, safety and tolerability of 2 different subcutaneous regimes of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy (DMD) resulting from mutation that can be corrected by exon skipping induced by GSK2402968. GSK2402968 is a novel drug that works by ??skipping? the altered part of the DMD gene and allows the production of partially functional protein, potentially leading to a less severe form of the condition. The study aims to recruit 54 subjects. Subjects will be randomized to receive a continuous regime (6mg/kg GSK2402968 once weekly) or an intermittent regimen (6mg/kg GSK2402968 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). All subjects will receive 6 mg/kg GSK2402968 twice weekly for the first 3 weeks of treatment, before starting continuous or intermittent regime. For each regime, subjects will be randomized to receive GSK2402968 or placebo (2:1). The study will be fully blinded with respect to active drug and placebo in each group however the different dose regimes will not be fully blinded. Subjects will be treated for 48 weeks. Safety, efficacy and tolerability will be measured at intervals throughout the study. A first, interim efficacy analysis will be conducted when all subject have completed 24 weeks of dosing. All subjects will remain on study until the final efficacy and safety evaluation after 48 weeks of dosing. An Independent Data Monitoring Committee will evaluate study drug safety and tolerability. At the end of the treatment period, subjects who have completed the study will be followed up for safety and progress for at least 20 weeks after the last dose of study drug or may be entered into an extension study.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  10/H0906/29

• Date of REC Opinion

  17 Jun 2010

• REC opinion

  Further Information Favourable Opinion