Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Muscular Dystrophies Clinical Trial

Official title:

An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01480245
• Other study ID #: 114349
• Status: Terminated
• Phase: Phase 3
• First received: November 23, 2011
• Last updated: March 21, 2017
• Start date: September 2011
• Est. completion date: March 2014

Study information

• Verified date: March 2017
• Source: GlaxoSmithKline
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 233
• Est. completion date: March 2014
• Est. primary completion date: March 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• GSK2402968
6mg/kg/week

Locations

Country	Name	City	State
Argentina	GSK Investigational Site	Ciudad Autónoma de Buenos Aires	Buenos Aires
Australia	GSK Investigational Site	Parkville	Victoria
Australia	GSK Investigational Site	WEstmead	New South Wales
Belgium	GSK Investigational Site	Gent
Belgium	GSK Investigational Site	Leuven
Brazil	GSK Investigational Site	Curitiba	Paraná
Brazil	GSK Investigational Site	Porto Alegre	Rio Grande Do Sul
Brazil	GSK Investigational Site	Ribeirao Preto	São Paulo
Brazil	GSK Investigational Site	Rio de Janeiro
Brazil	GSK Investigational Site	Santo Andre	São Paulo
Brazil	GSK Investigational Site	Sao Paulo	São Paulo
Bulgaria	GSK Investigational Site	Sofia
Canada	GSK Investigational Site	London	Ontario
Canada	GSK Investigational Site	Montreal	Quebec
Canada	GSK Investigational Site	Vancouver	British Columbia
Chile	GSK Investigational Site	Santiago	Región Metro De Santiago
Chile	GSK Investigational Site	Santiago
Chile	GSK Investigational Site	Temuco	Región De La Araucania
Czech Republic	GSK Investigational Site	Brno
Czech Republic	GSK Investigational Site	Praha 5
Denmark	GSK Investigational Site	Koebenhavn Oe
France	GSK Investigational Site	Bordeaux cedex
France	GSK Investigational Site	Lille cedex
France	GSK Investigational Site	Marseille cedex 5
France	GSK Investigational Site	Montpellier cedex 5
France	GSK Investigational Site	Nantes cedex 01
France	GSK Investigational Site	Paris
France	GSK Investigational Site	Paris cedex 15
France	GSK Investigational Site	Pau cedex
France	GSK Investigational Site	Toulouse cedex 9
Germany	GSK Investigational Site	Essen	Nordrhein-Westfalen
Germany	GSK Investigational Site	Freiburg	Baden-Wuerttemberg
Germany	GSK Investigational Site	Goettingen	Niedersachsen
Germany	GSK Investigational Site	Kiel	Schleswig-Holstein
Germany	GSK Investigational Site	Muenchen	Bayern
Hungary	GSK Investigational Site	Budapest
Israel	GSK Investigational Site	Jerusalem
Italy	GSK Investigational Site	Ferrara	Emilia-Romagna
Italy	GSK Investigational Site	Messina	Sicilia
Italy	GSK Investigational Site	Milano	Lombardia
Italy	GSK Investigational Site	Roma	Lazio
Italy	GSK Investigational Site	Roma	Lazio
Japan	GSK Investigational Site	Hyogo
Japan	GSK Investigational Site	Kumamoto
Japan	GSK Investigational Site	Saitama
Japan	GSK Investigational Site	Tokyo
Korea, Republic of	GSK Investigational Site	Seoul
Netherlands	GSK Investigational Site	Leiden
Netherlands	GSK Investigational Site	Nijmegen
Norway	GSK Investigational Site	Oslo
Poland	GSK Investigational Site	Warszawa
Russian Federation	GSK Investigational Site	Moscow
Spain	GSK Investigational Site	Esplugues de Llobregat. Barcelona
Spain	GSK Investigational Site	Madrid
Spain	GSK Investigational Site	Valencia
Taiwan	GSK Investigational Site	Kaohsiung
Turkey	GSK Investigational Site	Ankara
United Kingdom	GSK Investigational Site	London
United Kingdom	GSK Investigational Site	Newcastle-upon-Tyne

Sponsors (1)

Lead Sponsor	Collaborator
GlaxoSmithKline

Countries where clinical trial is conducted

Argentina,  Australia,  Belgium,  Brazil,  Bulgaria,  Canada,  Chile,  Czech Republic,  Denmark,  France,  Germany,  Hungary,  Israel,  Italy,  Japan,  Korea, Republic of,  Netherlands,  Norway,  Poland,  Russian Federation,  Spain,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Differences between the 6MWD at baseline and Week 104		104 weeks
Secondary	Timed Function tests		104 weeks
Secondary	Muscle strength		104 weeks
Secondary	North Star Ambulatory Assessment Scores		104 weeks
Secondary	Creatine kinase Serum concentrations		104 weeks
Secondary	Pulmonary Function		104 weeks
Secondary	Pediatric Quality of Life Neuromuscular module		104weeks
Secondary	Clinician Global Impression of Improvement		104 weeks
Secondary	Health Utilities Index		104 weeks
Secondary	Frequency of accidental falls during 6 Minute Walk Distance test		104 weeks
Secondary	Functional Outcomes Assessment		104 weeks
Secondary	Time to major disease milestones		104 weeks