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Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant 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: NCT01153932
Recruitment Status : Completed
First Posted : June 30, 2010
Last Update Posted : August 25, 2014
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline

Study Description
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Brief Summary:
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.

Condition or disease Intervention/treatment Phase
Muscular Dystrophies Drug: GSK2402968 Drug: matched placebo Phase 2

Detailed Description:
This is a phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study in ambulant subjects with DMD resulting from a mutation that can be corrected by exon skipping induced by GSK2402968. The study aims to randomise 54 subjects. There will be 2 parallel cohorts. Each cohort will include subjects on GSK2402968 and matched placebo in a 2:1 ratio.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 53 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy
Study Start Date : September 2010
Actual Primary Completion Date : March 2012
Actual Study Completion Date : September 2012

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Continuous regimen; 6mg/kg once weekly
Once Weekly
 Drug: GSK2402968
Subcutaneous injection
Other Name: 968

Drug: matched placebo
Subcutaneous injection
Other Name: Placebo
Experimental: Intermittent regimen; 6mg/kg twice weekly
Twice weekly on 1st, 3rd and 5th weeks, once weekly on 2nd, 4th and 6th weeks, and no active drug on 7th to 10th week of each 10 week cycle
 Drug: GSK2402968
Subcutaneous injection
Other Name: 968

Drug: matched placebo
Subcutaneous injection
Other Name: Placebo


Outcome Measures
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Primary Outcome Measures :
  1. To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD [ Time Frame: 48 weeks ]

Secondary Outcome Measures :
  1. To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects [ Time Frame: one year ]
  2. To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD [ Time Frame: 48 weeks ]
  3. To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD [ Time Frame: one year ]

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:

  • Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a state-of-the-art DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by GSK2402968-induced DMD exon 51 skipping,
  • Males, at least 5 years of age and with a life expectancy of at least 1 year
  • Able to rise from floor in ≤7 seconds (without aids/orthoses),
  • Able to complete the 6MWD test with a distance of at least 75m
  • Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study
  • QTc <450msec
  • On adequate contraception
  • Able to comply with and complete all protocol requirements

Exclusion Criteria:

  • any additional missing exon for DMD
  • Current of history of liver or renal disease or impairment
  • Acute illness within 4 weeks of the first dose
  • Use of prohibited meds within 6 months of fist dose
  • Current participation in any other investigational clinical trial
  • Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening
  • Symptomatic cardiomyopathy
  • Children in Care
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): NCT01153932


Locations
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Australia, New South Wales
GSK Investigational Site
WEstmead, New South Wales, Australia, 2145
Australia, Victoria
GSK Investigational Site
Parkville, Victoria, Australia, 3052
Belgium
GSK Investigational Site
Gent, Belgium, 9000
France
GSK Investigational Site
Paris cedex 13, France, 75651
Germany
GSK Investigational Site
Freiburg, Baden-Wuerttemberg, Germany, 79106
GSK Investigational Site
Essen, Nordrhein-Westfalen, Germany, 45122
Israel
GSK Investigational Site
Jerusalem, Israel, 91240
Netherlands
GSK Investigational Site
Nijmegen, Netherlands, 6525 GA
Spain
GSK Investigational Site
Esplugues (Barcelona), Spain, 08950
GSK Investigational Site
Valencia, Spain, 46009
Turkey
GSK Investigational Site
Ankara, Turkey, 06100
United Kingdom
GSK Investigational Site
London, United Kingdom, WC1N 1EH
GSK Investigational Site
Newcastle upon Tyne, United Kingdom, NE1 3BZ
Sponsors and Collaborators
GlaxoSmithKline
Investigators
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Study Director: GSK Clinical Trials GlaxoSmithKline
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01153932    
Other Study ID Numbers: 114117
First Posted: June 30, 2010    Key Record Dates
Last Update Posted: August 25, 2014
Last Verified: August 2013
Keywords provided by GlaxoSmithKline:
968
muscular dystrophy
duchenne
DMD
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