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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT01480245
Recruitment Status : Terminated (The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.)
First Posted : November 28, 2011
Last Update Posted : March 23, 2017
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline

November 23, 2011
November 28, 2011
March 23, 2017
September 2011
March 2014   (Final data collection date for primary outcome measure)
Differences between the 6MWD at baseline and Week 104 [ Time Frame: 104 weeks ]
Same as current
Complete list of historical versions of study NCT01480245 on ClinicalTrials.gov Archive Site
  • Timed Function tests [ Time Frame: 104 weeks ]
  • Muscle strength [ Time Frame: 104 weeks ]
  • 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 ]
Same as current
Not Provided
Not Provided
 
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
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.
Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Muscular Dystrophies
Drug: GSK2402968
6mg/kg/week
  • Experimental: Continuous Dosing
    GSK2402968 6mg/kg/week
    Intervention: Drug: GSK2402968
  • Experimental: Intermittent Dosing
    GSK2402968 6mg/kg/week
    Intervention: Drug: GSK2402968
  • No Intervention: Natural History Observation
    The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
233
220
March 2014
March 2014   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: Male
5 Years and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
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
 
 
NCT01480245
114349
No
Not Provided
Not Provided
GlaxoSmithKline
GlaxoSmithKline
Not Provided
Study Director: GSK Clinical Trials GlaxoSmithKline
GlaxoSmithKline
March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP