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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01480245




Registration number
NCT01480245
Ethics application status
Date submitted
23/11/2011
Date registered
28/11/2011
Date last updated
23/03/2017

Titles & IDs
Public title
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Scientific title
An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Secondary ID [1] 0 0
114349
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Muscular Dystrophies 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GSK2402968

Experimental: Continuous Dosing - GSK2402968 6mg/kg/week

Experimental: Intermittent Dosing - 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


Treatment: Drugs: GSK2402968
6mg/kg/week
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Differences between the 6MWD at baseline and Week 104
Timepoint [1] 0 0
104 weeks
Secondary outcome [1] 0 0
Timed Function tests
Timepoint [1] 0 0
104 weeks
Secondary outcome [2] 0 0
Muscle strength
Timepoint [2] 0 0
104 weeks
Secondary outcome [3] 0 0
North Star Ambulatory Assessment Scores
Timepoint [3] 0 0
104 weeks
Secondary outcome [4] 0 0
Creatine kinase Serum concentrations
Timepoint [4] 0 0
104 weeks
Secondary outcome [5] 0 0
Pulmonary Function
Timepoint [5] 0 0
104 weeks
Secondary outcome [6] 0 0
Pediatric Quality of Life Neuromuscular module
Timepoint [6] 0 0
104weeks
Secondary outcome [7] 0 0
Clinician Global Impression of Improvement
Timepoint [7] 0 0
104 weeks
Secondary outcome [8] 0 0
Health Utilities Index
Timepoint [8] 0 0
104 weeks
Secondary outcome [9] 0 0
Frequency of accidental falls during 6 Minute Walk Distance test
Timepoint [9] 0 0
104 weeks
Secondary outcome [10] 0 0
Functional Outcomes Assessment
Timepoint [10] 0 0
104 weeks
Secondary outcome [11] 0 0
Time to major disease milestones
Timepoint [11] 0 0
104 weeks

Eligibility
Key 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.
Minimum age
5 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/09/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/03/2014
Sample size
Target
Accrual to date
Final
233
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
GSK Investigational Site - WEstmead
Recruitment hospital [2] 0 0
GSK Investigational Site - Parkville
Recruitment postcode(s) [1] 0 0
2145 - WEstmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
Argentina
State/province [1] 0 0
Buenos Aires
Country [2] 0 0
Belgium
State/province [2] 0 0
Gent
Country [3] 0 0
Belgium
State/province [3] 0 0
Leuven
Country [4] 0 0
Brazil
State/province [4] 0 0
Paraná
Country [5] 0 0
Brazil
State/province [5] 0 0
Rio Grande Do Sul
Country [6] 0 0
Brazil
State/province [6] 0 0
São Paulo
Country [7] 0 0
Brazil
State/province [7] 0 0
Rio de Janeiro
Country [8] 0 0
Bulgaria
State/province [8] 0 0
Sofia
Country [9] 0 0
Canada
State/province [9] 0 0
British Columbia
Country [10] 0 0
Canada
State/province [10] 0 0
Ontario
Country [11] 0 0
Canada
State/province [11] 0 0
Quebec
Country [12] 0 0
Chile
State/province [12] 0 0
Región De La Araucania
Country [13] 0 0
Chile
State/province [13] 0 0
Región Metro De Santiago
Country [14] 0 0
Chile
State/province [14] 0 0
Santiago
Country [15] 0 0
Czech Republic
State/province [15] 0 0
Brno
Country [16] 0 0
Czech Republic
State/province [16] 0 0
Praha 5
Country [17] 0 0
Denmark
State/province [17] 0 0
Koebenhavn Oe
Country [18] 0 0
France
State/province [18] 0 0
Bordeaux cedex
Country [19] 0 0
France
State/province [19] 0 0
Lille cedex
Country [20] 0 0
France
State/province [20] 0 0
Marseille cedex 5
Country [21] 0 0
France
State/province [21] 0 0
Montpellier cedex 5
Country [22] 0 0
France
State/province [22] 0 0
Nantes cedex 01
Country [23] 0 0
France
State/province [23] 0 0
Paris cedex 15
Country [24] 0 0
France
State/province [24] 0 0
Paris
Country [25] 0 0
France
State/province [25] 0 0
Pau cedex
Country [26] 0 0
France
State/province [26] 0 0
Toulouse cedex 9
Country [27] 0 0
Germany
State/province [27] 0 0
Baden-Wuerttemberg
Country [28] 0 0
Germany
State/province [28] 0 0
Bayern
Country [29] 0 0
Germany
State/province [29] 0 0
Niedersachsen
Country [30] 0 0
Germany
State/province [30] 0 0
Nordrhein-Westfalen
Country [31] 0 0
Germany
State/province [31] 0 0
Schleswig-Holstein
Country [32] 0 0
Hungary
State/province [32] 0 0
Budapest
Country [33] 0 0
Israel
State/province [33] 0 0
Jerusalem
Country [34] 0 0
Italy
State/province [34] 0 0
Emilia-Romagna
Country [35] 0 0
Italy
State/province [35] 0 0
Lazio
Country [36] 0 0
Italy
State/province [36] 0 0
Lombardia
Country [37] 0 0
Italy
State/province [37] 0 0
Sicilia
Country [38] 0 0
Japan
State/province [38] 0 0
Hyogo
Country [39] 0 0
Japan
State/province [39] 0 0
Kumamoto
Country [40] 0 0
Japan
State/province [40] 0 0
Saitama
Country [41] 0 0
Japan
State/province [41] 0 0
Tokyo
Country [42] 0 0
Korea, Republic of
State/province [42] 0 0
Seoul
Country [43] 0 0
Netherlands
State/province [43] 0 0
Leiden
Country [44] 0 0
Netherlands
State/province [44] 0 0
Nijmegen
Country [45] 0 0
Norway
State/province [45] 0 0
Oslo
Country [46] 0 0
Poland
State/province [46] 0 0
Warszawa
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Moscow
Country [48] 0 0
Spain
State/province [48] 0 0
Esplugues de Llobregat. Barcelona
Country [49] 0 0
Spain
State/province [49] 0 0
Madrid
Country [50] 0 0
Spain
State/province [50] 0 0
Valencia
Country [51] 0 0
Taiwan
State/province [51] 0 0
Kaohsiung
Country [52] 0 0
Turkey
State/province [52] 0 0
Ankara
Country [53] 0 0
United Kingdom
State/province [53] 0 0
London
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Newcastle-upon-Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
GlaxoSmithKline
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT01480245
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
GSK Clinical Trials
Address 0 0
GlaxoSmithKline
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01480245