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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date  ICMJE November 23, 2011
First Posted Date  ICMJE November 28, 2011
Last Update Posted Date March 23, 2017
Study Start Date  ICMJE September 2011
Actual Primary Completion Date March 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 23, 2011)
Differences between the 6MWD at baseline and Week 104 [ Time Frame: 104 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 23, 2011)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophies
Intervention  ICMJE Drug: GSK2402968
6mg/kg/week
Study Arms  ICMJE
  • 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
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: September 25, 2014)
233
Original Estimated Enrollment  ICMJE
 (submitted: November 23, 2011)
220
Actual Study Completion Date  ICMJE March 2014
Actual Primary Completion Date March 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE 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
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01480245
Other Study ID Numbers  ICMJE 114349
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party GlaxoSmithKline
Study Sponsor  ICMJE GlaxoSmithKline
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: GSK Clinical Trials GlaxoSmithKline
PRS Account GlaxoSmithKline
Verification Date March 2017

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