Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

March 21, 2017 updated by: GlaxoSmithKline

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

233

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Buenos Aires
      • Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina, C1125ABD
        • GSK Investigational Site
  • Australia
    • New South Wales
      • WEstmead, New South Wales, Australia, 2145
        • GSK Investigational Site
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • GSK Investigational Site
  • Belgium
      • Gent, Belgium, 9000
        • GSK Investigational Site
      • Leuven, Belgium, 3000
        • GSK Investigational Site
  • Brazil
      • Rio de Janeiro, Brazil, 21941-490
        • GSK Investigational Site
    • Paraná
      • Curitiba, Paraná, Brazil, 80250-060
        • GSK Investigational Site
    • Rio Grande Do Sul
      • Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
        • GSK Investigational Site
    • São Paulo
      • 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
  • Bulgaria
      • Sofia, Bulgaria, 1431
        • GSK Investigational Site
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • GSK Investigational Site
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • GSK Investigational Site
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • GSK Investigational Site
  • Chile
      • Santiago, Chile, 8330074
        • GSK Investigational Site
    • Región De La Araucania
      • Temuco, Región De La Araucania, Chile
        • GSK Investigational Site
    • Región Metro De Santiago
      • Santiago, Región Metro De Santiago, Chile, 7500539
        • GSK Investigational Site
  • Czech Republic
      • Brno, Czech Republic, 613 00
        • GSK Investigational Site
      • Praha 5, Czech Republic
        • GSK Investigational Site
  • Denmark
      • Koebenhavn Oe, Denmark, 2100
        • GSK Investigational Site
  • France
      • 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, France, 75013
        • GSK Investigational Site
      • Paris cedex 15, France, 75743
        • GSK Investigational Site
      • Pau cedex, France, 64046
        • GSK Investigational Site
      • Toulouse cedex 9, France, 31059
        • GSK Investigational Site
  • Germany
    • Baden-Wuerttemberg
      • Freiburg, Baden-Wuerttemberg, Germany, 79106
        • GSK Investigational Site
    • Bayern
      • Muenchen, Bayern, Germany, 80337
        • GSK Investigational Site
    • Niedersachsen
      • Goettingen, Niedersachsen, Germany, 37075
        • GSK Investigational Site
    • Nordrhein-Westfalen
      • Essen, Nordrhein-Westfalen, Germany, 45122
        • GSK Investigational Site
    • Schleswig-Holstein
      • Kiel, Schleswig-Holstein, Germany, 24105
        • GSK Investigational Site
  • Hungary
      • Budapest, Hungary, 1095
        • GSK Investigational Site
  • Israel
      • Jerusalem, Israel, 91240
        • GSK Investigational Site
  • Italy
    • Emilia-Romagna
      • Ferrara, Emilia-Romagna, Italy, 44100
        • GSK Investigational Site
    • Lazio
      • Roma, Lazio, Italy, 00165
        • GSK Investigational Site
      • Roma, Lazio, Italy, 00168
        • GSK Investigational Site
    • Lombardia
      • Milano, Lombardia, Italy, 20122
        • GSK Investigational Site
    • Sicilia
      • Messina, Sicilia, Italy, 98125
        • GSK Investigational Site
  • Japan
      • 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
        • GSK Investigational Site
  • Korea, Republic of
      • Seoul, Korea, Republic of, 110-744
        • GSK Investigational Site
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • GSK Investigational Site
      • Nijmegen, Netherlands, 6525 GC
        • GSK Investigational Site
  • Norway
      • Oslo, Norway, 0027
        • GSK Investigational Site
  • Poland
      • Warszawa, Poland, 02-097
        • GSK Investigational Site
  • Russian Federation
      • Moscow, Russian Federation, 125412
        • GSK Investigational Site
  • Spain
      • Esplugues de Llobregat. Barcelona, Spain, 08950
        • GSK Investigational Site
      • Madrid, Spain, 28046
        • GSK Investigational Site
      • Valencia, Spain, 46026
        • GSK Investigational Site
  • Taiwan
      • Kaohsiung, Taiwan, 80708
        • GSK Investigational Site
  • Turkey
      • Ankara, Turkey, 06100
        • GSK Investigational Site
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • GSK Investigational Site
      • Newcastle-upon-Tyne, United Kingdom, NE1 4LP
        • GSK Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Differences between the 6MWD at baseline and Week 104
Time Frame: 104 weeks
104 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Timed Function tests
Time Frame: 104 weeks
104 weeks
Muscle strength
Time Frame: 104 weeks
104 weeks
North Star Ambulatory Assessment Scores
Time Frame: 104 weeks
104 weeks
Creatine kinase Serum concentrations
Time Frame: 104 weeks
104 weeks
Pulmonary Function
Time Frame: 104 weeks
104 weeks
Pediatric Quality of Life Neuromuscular module
Time Frame: 104weeks
104weeks
Clinician Global Impression of Improvement
Time Frame: 104 weeks
104 weeks
Health Utilities Index
Time Frame: 104 weeks
104 weeks
Frequency of accidental falls during 6 Minute Walk Distance test
Time Frame: 104 weeks
104 weeks
Functional Outcomes Assessment
Time Frame: 104 weeks
104 weeks
Time to major disease milestones
Time Frame: 104 weeks
104 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

GlaxoSmithKline

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2011

Primary Completion (ACTUAL)

March 1, 2014

Study Completion (ACTUAL)

March 1, 2014

Study Registration Dates

First Submitted

November 23, 2011

First Submitted That Met QC Criteria

November 23, 2011

First Posted (ESTIMATE)

November 28, 2011

Study Record Updates

Last Update Posted (ACTUAL)

March 23, 2017

Last Update Submitted That Met QC Criteria

March 21, 2017

Last Verified

March 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 114349

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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