Status:
COMPLETED
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
Lead Sponsor:
GlaxoSmithKline
Conditions:
Muscular Dystrophies
Eligibility:
MALE
5+ years
Phase:
PHASE2
Brief Summary
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
Detailed Description
This is a phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study in ambulant subjects with DMD resulting from a mutation that can be corrected by exon skipping induced ...
Eligibility Criteria
Inclusion
- Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation in 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), CGH (Comparative Genomic Hybridisation) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by GSK2402968-induced DMD exon 51 skipping,
- Males, at least 5 years of age and with a life expectancy of at least 1 year
- Able to rise from floor in ≤7 seconds (without aids/orthoses),
- Able to complete the 6MWD test with a distance of at least 75m
- Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study
- QTc \<450msec
- On adequate contraception
- Able to comply with and complete all protocol requirements
Exclusion
- any additional missing exon for DMD
- Current of history of liver or renal disease or impairment
- Acute illness within 4 weeks of the first dose
- Use of prohibited meds within 6 months of fist dose
- Current participation in any other investigational clinical trial
- Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening
- Symptomatic cardiomyopathy
- Children in Care
Key Trial Info
Start Date :
September 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2012
Estimated Enrollment :
53 Patients enrolled
Trial Details
Trial ID
NCT01153932
Start Date
September 1 2010
End Date
September 1 2012
Last Update
August 25 2014
Active Locations (13)
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1
GSK Investigational Site
Westmead, New South Wales, Australia, 2145
2
GSK Investigational Site
Parkville, Victoria, Australia, 3052
3
GSK Investigational Site
Ghent, Belgium, 9000
4
GSK Investigational Site
Paris, France, 75651