Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Phase 3

Terminated

• Conditions: Muscular Dystrophies
• Interventions: Drug: GSK2402968

• Registration Number: NCT01480245
• Lead Sponsor: GlaxoSmithKline

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-09
• Study First Submitted: 2011-11-23
• Study First Submitted QC: 2011-11-23
• Study First Posted: 2011-11-28
• Primary Completion: 2014-03
• Study Completion: 2014-03
• Status Verified: 2017-03
• Last Update Submitted: 2017-03-21
• Last Update Posted: 2017-03-23

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 233

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Continuous Dosing	GSK2402968	GSK2402968 6mg/kg/week
Intermittent Dosing	GSK2402968	GSK2402968 6mg/kg/week

Primary Outcome Measures

Name	Time	Method
Differences between the 6MWD at baseline and Week 104	104 weeks

Secondary Outcome Measures

Name	Time	Method
Pediatric Quality of Life Neuromuscular module	104weeks
Time to major disease milestones	104 weeks
Timed Function tests	104 weeks
Muscle strength	104 weeks
North Star Ambulatory Assessment Scores	104 weeks
Functional Outcomes Assessment	104 weeks
Creatine kinase Serum concentrations	104 weeks
Pulmonary Function	104 weeks
Frequency of accidental falls during 6 Minute Walk Distance test	104 weeks
Clinician Global Impression of Improvement	104 weeks
Health Utilities Index	104 weeks

Trial Locations

Locations (1)

GSK Investigational Site; 🇬🇧 Newcastle-upon-Tyne, United Kingdom