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

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
233

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Differences between the 6MWD at baseline and Week 104
Time Frame: 104 weeks
104 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 1, 2011

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2014

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 1, 2014

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 23, 2011

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 23, 2011

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 28, 2011

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 23, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 21, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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