Clinical trial NCT01480245: Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Official title: An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Muscular Dystrophies
Keywords: 968
Duchenne
DMD
drisapersen
Lead sponsor: GlaxoSmithKline
Sponsor class: INDUSTRY
First posted: 2011-11-28
Enrollment: 233
Sex: MALE
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Differences between the 6MWD at baseline and Week 104
timeFrame: 104 weeks
Secondary outcomes:
measure: Timed Function tests
timeFrame: 104 weeks
measure: Muscle strength
timeFrame: 104 weeks
measure: North Star Ambulatory Assessment Scores
timeFrame: 104 weeks
measure: Creatine kinase Serum concentrations
timeFrame: 104 weeks
measure: Pulmonary Function
timeFrame: 104 weeks
measure: Pediatric Quality of Life Neuromuscular module
timeFrame: 104weeks
measure: Clinician Global Impression of Improvement
timeFrame: 104 weeks
measure: Health Utilities Index
timeFrame: 104 weeks
measure: Frequency of accidental falls during 6 Minute Walk Distance test
timeFrame: 104 weeks
measure: Functional Outcomes Assessment
timeFrame: 104 weeks
measure: Time to major disease milestones
timeFrame: 104 weeks
Other outcomes:
Locations:
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: GSK Investigational Site
state: Buenos Aires
city: WEstmead
country: Australia
facility: GSK Investigational Site
state: New South Wales
city: Parkville
country: Australia
facility: GSK Investigational Site
state: Victoria
city: Gent
country: Belgium
facility: GSK Investigational Site
city: Leuven
country: Belgium
facility: GSK Investigational Site
city: Curitiba
country: Brazil
facility: GSK Investigational Site
state: Paraná
city: Porto Alegre
country: Brazil
facility: GSK Investigational Site
state: Rio Grande Do Sul
city: Ribeirao Preto
country: Brazil
facility: GSK Investigational Site
state: São Paulo
city: Santo Andre
country: Brazil
facility: GSK Investigational Site
state: São Paulo
city: Sao Paulo
country: Brazil
facility: GSK Investigational Site
state: São Paulo
city: Rio de Janeiro
country: Brazil
facility: GSK Investigational Site
city: Sofia
country: Bulgaria
facility: GSK Investigational Site
city: Vancouver
country: Canada
facility: GSK Investigational Site
state: British Columbia
city: London
country: Canada
facility: GSK Investigational Site
state: Ontario
city: Montreal
country: Canada
facility: GSK Investigational Site
state: Quebec
city: Temuco
country: Chile
facility: GSK Investigational Site
state: Región De La Araucania
city: Santiago
country: Chile
facility: GSK Investigational Site
state: Región Metro De Santiago
city: Santiago
country: Chile
facility: GSK Investigational Site
city: Brno
country: Czech Republic
facility: GSK Investigational Site
city: Praha 5
country: Czech Republic
facility: GSK Investigational Site
city: Koebenhavn Oe
country: Denmark
facility: GSK Investigational Site
city: Bordeaux cedex
country: France
facility: GSK Investigational Site
city: Lille cedex
country: France
facility: GSK Investigational Site
city: Marseille cedex 5
country: France
facility: GSK Investigational Site
city: Montpellier cedex 5
country: France
facility: GSK Investigational Site
city: Nantes cedex 01
country: France
facility: GSK Investigational Site
city: Paris cedex 15
country: France
facility: GSK Investigational Site
city: Paris
country: France
facility: GSK Investigational Site
city: Pau cedex
country: France
facility: GSK Investigational Site
city: Toulouse cedex 9
country: France
facility: GSK Investigational Site
city: Freiburg
country: Germany
facility: GSK Investigational Site
state: Baden-Wuerttemberg
city: Muenchen
country: Germany
facility: GSK Investigational Site
state: Bayern
city: Goettingen
country: Germany
facility: GSK Investigational Site
state: Niedersachsen
city: Essen
country: Germany
facility: GSK Investigational Site
state: Nordrhein-Westfalen
city: Kiel
country: Germany
facility: GSK Investigational Site
state: Schleswig-Holstein
city: Budapest
country: Hungary
facility: GSK Investigational Site
city: Jerusalem
country: Israel
facility: GSK Investigational Site
city: Ferrara
country: Italy
facility: GSK Investigational Site
state: Emilia-Romagna
city: Roma
country: Italy
facility: GSK Investigational Site
state: Lazio
city: Roma
country: Italy
facility: GSK Investigational Site
state: Lazio
city: Milano
country: Italy
facility: GSK Investigational Site
state: Lombardia
city: Messina
country: Italy
facility: GSK Investigational Site
state: Sicilia
city: Hyogo
country: Japan
facility: GSK Investigational Site
city: Kumamoto
country: Japan
facility: GSK Investigational Site
city: Saitama
country: Japan
facility: GSK Investigational Site
city: Tokyo
country: Japan
facility: GSK Investigational Site
city: Seoul
country: Korea, Republic of
facility: GSK Investigational Site
city: Leiden
country: Netherlands
facility: GSK Investigational Site
city: Nijmegen
country: Netherlands
facility: GSK Investigational Site
city: Oslo
country: Norway
facility: GSK Investigational Site
city: Warszawa
country: Poland
facility: GSK Investigational Site
city: Moscow
country: Russian Federation
facility: GSK Investigational Site
city: Esplugues de Llobregat. Barcelona
country: Spain
facility: GSK Investigational Site
city: Madrid
country: Spain
facility: GSK Investigational Site
city: Valencia
country: Spain
facility: GSK Investigational Site
city: Kaohsiung
country: Taiwan
facility: GSK Investigational Site
city: Ankara
country: Turkey
facility: GSK Investigational Site
city: London
country: United Kingdom
facility: GSK Investigational Site
city: Newcastle-upon-Tyne
country: United Kingdom
facility: GSK Investigational Site
Study officials:
affiliation: GlaxoSmithKline
name: GSK Clinical Trials
role: STUDY_DIRECTOR
References: