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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

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 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


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



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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
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.

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


  Hide Study Locations
Locations
Argentina
GSK Investigational Site
Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina, C1125ABD
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
GSK Investigational Site
Leuven, Belgium, 3000
Brazil
GSK Investigational Site
Curitiba, Paraná, Brazil, 80250-060
GSK Investigational Site
Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
GSK Investigational Site
Ribeirao Preto, São Paulo, Brazil, 14048-900
GSK Investigational Site
Santo Andre, São Paulo, Brazil, 09060-650
GSK Investigational Site
Sao Paulo, São Paulo, Brazil, 05403-000
GSK Investigational Site
Rio de Janeiro, Brazil, 21941-490
Bulgaria
GSK Investigational Site
Sofia, Bulgaria, 1431
Canada, British Columbia
GSK Investigational Site
Vancouver, British Columbia, Canada, V6H 3V4
Canada, Ontario
GSK Investigational Site
London, Ontario, Canada, N6A 5W9
Canada, Quebec
GSK Investigational Site
Montreal, Quebec, Canada, H3T 1C5
Chile
GSK Investigational Site
Temuco, Región De La Araucania, Chile
GSK Investigational Site
Santiago, Región Metro De Santiago, Chile, 7500539
GSK Investigational Site
Santiago, Chile, 8330074
Czech Republic
GSK Investigational Site
Brno, Czech Republic, 613 00
GSK Investigational Site
Praha 5, Czech Republic
Denmark
GSK Investigational Site
Koebenhavn Oe, Denmark, 2100
France
GSK Investigational Site
Bordeaux cedex, France, 33076
GSK Investigational Site
Lille cedex, France, 59037
GSK Investigational Site
Marseille cedex 5, France, 13385
GSK Investigational Site
Montpellier cedex 5, France, 34295
GSK Investigational Site
Nantes cedex 01, France, 44093
GSK Investigational Site
Paris cedex 15, France, 75743
GSK Investigational Site
Paris, France, 75013
GSK Investigational Site
Pau cedex, France, 64046
GSK Investigational Site
Toulouse cedex 9, France, 31059
Germany
GSK Investigational Site
Freiburg, Baden-Wuerttemberg, Germany, 79106
GSK Investigational Site
Muenchen, Bayern, Germany, 80337
GSK Investigational Site
Goettingen, Niedersachsen, Germany, 37075
GSK Investigational Site
Essen, Nordrhein-Westfalen, Germany, 45122
GSK Investigational Site
Kiel, Schleswig-Holstein, Germany, 24105
Hungary
GSK Investigational Site
Budapest, Hungary, 1095
Israel
GSK Investigational Site
Jerusalem, Israel, 91240
Italy
GSK Investigational Site
Ferrara, Emilia-Romagna, Italy, 44100
GSK Investigational Site
Roma, Lazio, Italy, 00165
GSK Investigational Site
Roma, Lazio, Italy, 00168
GSK Investigational Site
Milano, Lombardia, Italy, 20122
GSK Investigational Site
Messina, Sicilia, Italy, 98125
Japan
GSK Investigational Site
Hyogo, Japan, 650-0017
GSK Investigational Site
Kumamoto, Japan, 860-8556
GSK Investigational Site
Saitama, Japan, 349-0196
GSK Investigational Site
Tokyo, Japan, 187-8551
Korea, Republic of
GSK Investigational Site
Seoul, Korea, Republic of, 110-744
Netherlands
GSK Investigational Site
Leiden, Netherlands, 2333 ZA
GSK Investigational Site
Nijmegen, Netherlands, 6525 GC
Norway
GSK Investigational Site
Oslo, Norway, 0027
Poland
GSK Investigational Site
Warszawa, Poland, 02-097
Russian Federation
GSK Investigational Site
Moscow, Russian Federation, 125412
Spain
GSK Investigational Site
Esplugues de Llobregat. Barcelona, Spain, 08950
GSK Investigational Site
Madrid, Spain, 28046
GSK Investigational Site
Valencia, Spain, 46026
Taiwan
GSK Investigational Site
Kaohsiung, Taiwan, 80708
Turkey
GSK Investigational Site
Ankara, Turkey, 06100
United Kingdom
GSK Investigational Site
London, United Kingdom, WC1N 3JH
GSK Investigational Site
Newcastle-upon-Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01480245     History of Changes
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:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked