Muscular Dystrophies
Conditions
Keywords
968, Duchenne, DMD, drisapersen
Brief summary
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.
Interventions
DRUGGSK2402968
6mg/kg/week
Sponsors
GlaxoSmithKline
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Differences between the 6MWD at baseline and Week 104 | 104 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Timed Function tests | 104 weeks |
| Muscle strength | 104 weeks |
| North Star Ambulatory Assessment Scores | 104 weeks |
| Creatine kinase Serum concentrations | 104 weeks |
| Pulmonary Function | 104 weeks |
| Time to major disease milestones | 104 weeks |
| Clinician Global Impression of Improvement | 104 weeks |
| Health Utilities Index | 104 weeks |
| Frequency of accidental falls during 6 Minute Walk Distance test | 104 weeks |
| Functional Outcomes Assessment | 104 weeks |
| Pediatric Quality of Life Neuromuscular module | 104weeks |
Countries
Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Netherlands, Norway, Poland, Russia, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom
Outcome results
None listed