Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01480245
• Date of registration: 23/11/2011
• Prospective Registration: No
• Primary sponsor: GlaxoSmithKline
• Public title: Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
• Scientific title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
• Date of first enrolment: September 2011
• Target sample size: 233
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01480245
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Korea, Republic of, Netherlands, Norway, Poland, Russian Federation, Spain, Taiwan, Turkey, United Kingdom

Contacts

• Name: GSK Clinical Trials
• Affiliation: GlaxoSmithKline

Key inclusion & exclusion criteria

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.



Age minimum: 5 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophies

Intervention(s)

Drug: GSK2402968

Primary Outcome(s)

Differences between the 6MWD at baseline and Week 104 [Time Frame: 104 weeks]

Secondary Outcome(s)

Time to major disease milestones [Time Frame: 104 weeks]

Functional Outcomes Assessment [Time Frame: 104 weeks]

Health Utilities Index [Time Frame: 104 weeks]

North Star Ambulatory Assessment Scores [Time Frame: 104 weeks]

Frequency of accidental falls during 6 Minute Walk Distance test [Time Frame: 104 weeks]

Muscle strength [Time Frame: 104 weeks]

Pediatric Quality of Life Neuromuscular module [Time Frame: 104weeks]

Pulmonary Function [Time Frame: 104 weeks]

Creatine kinase Serum concentrations [Time Frame: 104 weeks]

Clinician Global Impression of Improvement [Time Frame: 104 weeks]

Timed Function tests [Time Frame: 104 weeks]

Secondary ID(s)

114349

Source(s) of Monetary Support

Please refer to primary and secondary sponsors