Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Study Overview
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Brief Summary
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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.

Official Title
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An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Conditions
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Muscular Dystrophies

Intervention / Treatment
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* Drug: GSK2402968


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Continuous Dosing<br>GSK2402968 6mg/kg/week | Drug: GSK2402968<br>* 6mg/kg/week<br>|
| Experimental: Intermittent Dosing<br>GSK2402968 6mg/kg/week | Drug: GSK2402968<br>* 6mg/kg/week<br>|
| No Intervention: Natural History Observation<br>The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Differences between the 6MWD at baseline and Week 104 |  | 104 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | 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 | 
| Pediatric Quality of Life Neuromuscular module |  | 104weeks | 
| 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 | 
| Time to major disease milestones |  | 104 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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968, Duchenne, DMD, drisapersen