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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Primary Purpose

Muscular Dystrophies

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
GSK2402968
Sponsored by
GlaxoSmithKline
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophies focused on measuring 968, Duchenne, DMD, drisapersen

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

No Intervention

Arm Label

Continuous Dosing

Intermittent Dosing

Natural History Observation

Arm Description

GSK2402968 6mg/kg/week

GSK2402968 6mg/kg/week

The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment

Outcomes

Primary Outcome Measures

Differences between the 6MWD at baseline and Week 104

Secondary Outcome Measures

Timed Function tests
Muscle strength
North Star Ambulatory Assessment Scores
Creatine kinase Serum concentrations
Pulmonary Function
Pediatric Quality of Life Neuromuscular module
Clinician Global Impression of Improvement
Health Utilities Index
Frequency of accidental falls during 6 Minute Walk Distance test
Functional Outcomes Assessment
Time to major disease milestones

Full Information

First Posted
November 23, 2011
Last Updated
March 21, 2017
Sponsor
GlaxoSmithKline
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1. Study Identification

Unique Protocol Identification Number
NCT01480245
Brief Title
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

2. Study Status

Record Verification Date
March 2017
Overall Recruitment Status
Terminated
Why Stopped
The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.
Study Start Date
September 2011 (undefined)
Primary Completion Date
March 2014 (Actual)
Study Completion Date
March 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
GlaxoSmithKline

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophies
Keywords
968, Duchenne, DMD, drisapersen

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
233 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Continuous Dosing
Arm Type
Experimental
Arm Description
GSK2402968 6mg/kg/week
Arm Title
Intermittent Dosing
Arm Type
Experimental
Arm Description
GSK2402968 6mg/kg/week
Arm Title
Natural History Observation
Arm Type
No Intervention
Arm Description
The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment
Intervention Type
Drug
Intervention Name(s)
GSK2402968
Intervention Description
6mg/kg/week
Primary Outcome Measure Information:
Title
Differences between the 6MWD at baseline and Week 104
Time Frame
104 weeks
Secondary Outcome Measure Information:
Title
Timed Function tests
Time Frame
104 weeks
Title
Muscle strength
Time Frame
104 weeks
Title
North Star Ambulatory Assessment Scores
Time Frame
104 weeks
Title
Creatine kinase Serum concentrations
Time Frame
104 weeks
Title
Pulmonary Function
Time Frame
104 weeks
Title
Pediatric Quality of Life Neuromuscular module
Time Frame
104weeks
Title
Clinician Global Impression of Improvement
Time Frame
104 weeks
Title
Health Utilities Index
Time Frame
104 weeks
Title
Frequency of accidental falls during 6 Minute Walk Distance test
Time Frame
104 weeks
Title
Functional Outcomes Assessment
Time Frame
104 weeks
Title
Time to major disease milestones
Time Frame
104 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
GSK Clinical Trials
Organizational Affiliation
GlaxoSmithKline
Official's Role
Study Director
Facility Information:
Facility Name
GSK Investigational Site
City
Ciudad Autónoma de Buenos Aires
State/Province
Buenos Aires
ZIP/Postal Code
C1125ABD
Country
Argentina
Facility Name
GSK Investigational Site
City
WEstmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
GSK Investigational Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
GSK Investigational Site
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
GSK Investigational Site
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
GSK Investigational Site
City
Curitiba
State/Province
Paraná
ZIP/Postal Code
80250-060
Country
Brazil
Facility Name
GSK Investigational Site
City
Porto Alegre
State/Province
Rio Grande Do Sul
ZIP/Postal Code
90035-903
Country
Brazil
Facility Name
GSK Investigational Site
City
Ribeirao Preto
State/Province
São Paulo
ZIP/Postal Code
14048-900
Country
Brazil
Facility Name
GSK Investigational Site
City
Santo Andre
State/Province
São Paulo
ZIP/Postal Code
09060-650
Country
Brazil
Facility Name
GSK Investigational Site
City
Sao Paulo
State/Province
São Paulo
ZIP/Postal Code
05403-000
Country
Brazil
Facility Name
GSK Investigational Site
City
Rio de Janeiro
ZIP/Postal Code
21941-490
Country
Brazil
Facility Name
GSK Investigational Site
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
GSK Investigational Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
GSK Investigational Site
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
GSK Investigational Site
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
GSK Investigational Site
City
Temuco
State/Province
Región De La Araucania
Country
Chile
Facility Name
GSK Investigational Site
City
Santiago
State/Province
Región Metro De Santiago
ZIP/Postal Code
7500539
Country
Chile
Facility Name
GSK Investigational Site
City
Santiago
ZIP/Postal Code
8330074
Country
Chile
Facility Name
GSK Investigational Site
City
Brno
ZIP/Postal Code
613 00
Country
Czech Republic
Facility Name
GSK Investigational Site
City
Praha 5
Country
Czech Republic
Facility Name
GSK Investigational Site
City
Koebenhavn Oe
ZIP/Postal Code
2100
Country
Denmark
Facility Name
GSK Investigational Site
City
Bordeaux cedex
ZIP/Postal Code
33076
Country
France
Facility Name
GSK Investigational Site
City
Lille cedex
ZIP/Postal Code
59037
Country
France
Facility Name
GSK Investigational Site
City
Marseille cedex 5
ZIP/Postal Code
13385
Country
France
Facility Name
GSK Investigational Site
City
Montpellier cedex 5
ZIP/Postal Code
34295
Country
France
Facility Name
GSK Investigational Site
City
Nantes cedex 01
ZIP/Postal Code
44093
Country
France
Facility Name
GSK Investigational Site
City
Paris cedex 15
ZIP/Postal Code
75743
Country
France
Facility Name
GSK Investigational Site
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
GSK Investigational Site
City
Pau cedex
ZIP/Postal Code
64046
Country
France
Facility Name
GSK Investigational Site
City
Toulouse cedex 9
ZIP/Postal Code
31059
Country
France
Facility Name
GSK Investigational Site
City
Freiburg
State/Province
Baden-Wuerttemberg
ZIP/Postal Code
79106
Country
Germany
Facility Name
GSK Investigational Site
City
Muenchen
State/Province
Bayern
ZIP/Postal Code
80337
Country
Germany
Facility Name
GSK Investigational Site
City
Goettingen
State/Province
Niedersachsen
ZIP/Postal Code
37075
Country
Germany
Facility Name
GSK Investigational Site
City
Essen
State/Province
Nordrhein-Westfalen
ZIP/Postal Code
45122
Country
Germany
Facility Name
GSK Investigational Site
City
Kiel
State/Province
Schleswig-Holstein
ZIP/Postal Code
24105
Country
Germany
Facility Name
GSK Investigational Site
City
Budapest
ZIP/Postal Code
1095
Country
Hungary
Facility Name
GSK Investigational Site
City
Jerusalem
ZIP/Postal Code
91240
Country
Israel
Facility Name
GSK Investigational Site
City
Ferrara
State/Province
Emilia-Romagna
ZIP/Postal Code
44100
Country
Italy
Facility Name
GSK Investigational Site
City
Roma
State/Province
Lazio
ZIP/Postal Code
00165
Country
Italy
Facility Name
GSK Investigational Site
City
Roma
State/Province
Lazio
ZIP/Postal Code
00168
Country
Italy
Facility Name
GSK Investigational Site
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20122
Country
Italy
Facility Name
GSK Investigational Site
City
Messina
State/Province
Sicilia
ZIP/Postal Code
98125
Country
Italy
Facility Name
GSK Investigational Site
City
Hyogo
ZIP/Postal Code
650-0017
Country
Japan
Facility Name
GSK Investigational Site
City
Kumamoto
ZIP/Postal Code
860-8556
Country
Japan
Facility Name
GSK Investigational Site
City
Saitama
ZIP/Postal Code
349-0196
Country
Japan
Facility Name
GSK Investigational Site
City
Tokyo
ZIP/Postal Code
187-8551
Country
Japan
Facility Name
GSK Investigational Site
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of
Facility Name
GSK Investigational Site
City
Leiden
ZIP/Postal Code
2333 ZA
Country
Netherlands
Facility Name
GSK Investigational Site
City
Nijmegen
ZIP/Postal Code
6525 GC
Country
Netherlands
Facility Name
GSK Investigational Site
City
Oslo
ZIP/Postal Code
0027
Country
Norway
Facility Name
GSK Investigational Site
City
Warszawa
ZIP/Postal Code
02-097
Country
Poland
Facility Name
GSK Investigational Site
City
Moscow
ZIP/Postal Code
125412
Country
Russian Federation
Facility Name
GSK Investigational Site
City
Esplugues de Llobregat. Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
GSK Investigational Site
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
GSK Investigational Site
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
GSK Investigational Site
City
Kaohsiung
ZIP/Postal Code
80708
Country
Taiwan
Facility Name
GSK Investigational Site
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
GSK Investigational Site
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
GSK Investigational Site
City
Newcastle-upon-Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

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