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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
study id #: NCT01480245
condition: Muscular Dystrophies
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
mechanism of action: Exon-skipping to promote dystrophin production
start date: September 2011
estimated completion: March 2014
phase of development: Phase 3
size / enrollment: 233
- Differences between the 6MWD at baseline and Week 104 [Time Frame:104 weeks]
- Timed Function tests [Time Frame: 104 weeks]
- Muscle strength [Time Frame: 104 week]
- North Star Ambulatory Assessment Scores [Time Frame: 104 weeks]
- Creatine kinase Serum concentrations [Time Frame: 104 weeks]
- Pulmonary Function [Time Frame: 104 weeks]
- Pediatric Quality of Life Neuromuscular module [Time Frame: 104weeks]
- Clinician Global Impression of Improvement [Time Frame: 104 weeks]
- Health Utilities Index [Time Frame: 104 weeks]
- Frequency of accidental falls during 6 Minute Walk Distance test [Time Frame: 104 weeks]
- Functional Outcomes Assessment [Time Frame: 104 weeks]
- Time to major disease milestones [Time Frame: 104 weeks]
• Previous participation in either DMD114117 or DMD114044
• Continued use of glucocorticoids
• Willing and able to comply with all protocol requirements
• Able to give informed consent
• French subjects: Eligible for inclusion only if either affiliated to or a beneficiary of a social security category.
• Subject experienced a serious adverse event or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in opinion of the investigator could have been attributable to study medication and is ongoing,
• Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, except GSK2402968, within 1 month of the first administration of study medication,
• Current or anticipated participation in any investigational clinical studies,
• History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current history of renal or liver disease/impairment, history of inflammatory disease.
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