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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Muscular Dystrophies Drug: GSK2402968 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 233 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Study Start Date : September 2011
Actual Primary Completion Date : March 2014
Actual Study Completion Date : March 2014

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Continuous Dosing
GSK2402968 6mg/kg/week
 Drug: GSK2402968
6mg/kg/week
Experimental: Intermittent Dosing
GSK2402968 6mg/kg/week
 Drug: GSK2402968
6mg/kg/week
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


Outcome Measures
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Primary Outcome Measures :
  1. Differences between the 6MWD at baseline and Week 104 [ Time Frame: 104 weeks ]

Secondary Outcome Measures :
  1. Timed Function tests [ Time Frame: 104 weeks ]
  2. Muscle strength [ Time Frame: 104 weeks ]
  3. North Star Ambulatory Assessment Scores [ Time Frame: 104 weeks ]
  4. Creatine kinase Serum concentrations [ Time Frame: 104 weeks ]
  5. Pulmonary Function [ Time Frame: 104 weeks ]
  6. Pediatric Quality of Life Neuromuscular module [ Time Frame: 104weeks ]
  7. Clinician Global Impression of Improvement [ Time Frame: 104 weeks ]
  8. Health Utilities Index [ Time Frame: 104 weeks ]
  9. Frequency of accidental falls during 6 Minute Walk Distance test [ Time Frame: 104 weeks ]
  10. Functional Outcomes Assessment [ Time Frame: 104 weeks ]
  11. Time to major disease milestones [ Time Frame: 104 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01480245


Locations
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Sponsors and Collaborators
GlaxoSmithKline
Investigators
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Study Director: GSK Clinical Trials GlaxoSmithKline
More Information
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Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01480245    
Other Study ID Numbers: 114349
First Posted: November 28, 2011    Key Record Dates
Last Update Posted: March 23, 2017
Last Verified: March 2017
Keywords provided by GlaxoSmithKline:
968
Duchenne
DMD
drisapersen
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked