Muscular DystrophiesPhase 3NCT01480245
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
A Phase 3 study testing GSK2402968 in people with Muscular Dystrophies, run by GlaxoSmithKline at 59 sites in 24 countries.
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. Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Muscular Dystrophies
- What is being tested?
- GSK2402968
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 233 participants
- Where
- 59 sitesin 24 countries
- Study dates
- Sep 2011to Mar 2014
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
59 sites in 24 countries.
59 sites in 24 countries
France9 sites
GSK Investigational Site
Bordeaux, 33076
GSK Investigational Site
Lille, 59037
GSK Investigational Site
Marseille, 13385
GSK Investigational Site
Montpellier, 34295
GSK Investigational Site
Nantes, 44093
GSK Investigational Site
Paris, 75013
GSK Investigational Site
Paris, 75743
GSK Investigational Site
Pau, 64046
GSK Investigational Site
Toulouse, 31059
Brazil6 sites
GSK Investigational Site
Curitiba, Paraná, 80250-060
GSK Investigational Site
Porto Alegre, Rio Grande do Sul, 90035-903
GSK Investigational Site
Ribeirão Preto, São Paulo, 14048-900
GSK Investigational Site
Rio de Janeiro, Rio de Janeiro, 21941-490
GSK Investigational Site
Santo André, São Paulo, 09060-650
GSK Investigational Site
São Paulo, São Paulo, 05403-000
Germany5 sites
GSK Investigational Site
Essen, North Rhine-Westphalia, 45122
GSK Investigational Site
Freiburg im Breisgau, Baden-Wurttemberg, 79106
GSK Investigational Site
Göttingen, Lower Saxony, 37075
GSK Investigational Site
Kiel, Schleswig-Holstein, 24105
GSK Investigational Site
Munich, Bavaria, 80337
Italy5 sites
GSK Investigational Site
Ferrara, Emilia-Romagna, 44100
GSK Investigational Site
Messina, Sicily, 98125
GSK Investigational Site
Milan, Lombardy, 20122
GSK Investigational Site
Rome, Lazio, 00165
GSK Investigational Site
Rome, Lazio, 00168
Japan4 sites
GSK Investigational Site
Hyōgo, 650-0017
GSK Investigational Site
Kumamoto, 860-8556
GSK Investigational Site
Saitama, 349-0196
GSK Investigational Site
Tokyo, 187-8551
Canada3 sites
GSK Investigational Site
London, Ontario, N6A 5W9
GSK Investigational Site
Montreal, Quebec, H3T 1C5
GSK Investigational Site
Vancouver, British Columbia, V6H 3V4
Chile3 sites
GSK Investigational Site
Santiago, Región Metro de Santiago, 7500539
GSK Investigational Site
Santiago, 8330074
GSK Investigational Site
Temuco, Región de La Araucania
Spain3 sites
GSK Investigational Site
Esplugues de Llobregat. Barcelona, 08950
GSK Investigational Site
Madrid, 28046
GSK Investigational Site
Valencia, 46026
Australia2 sites
GSK Investigational Site
Parkville, Victoria, 3052
GSK Investigational Site
Westmead, New South Wales, 2145
Belgium2 sites
GSK Investigational Site
Ghent, 9000
GSK Investigational Site
Leuven, 3000
Czechia2 sites
GSK Investigational Site
Brno, 613 00
GSK Investigational Site
Prague
Netherlands2 sites
GSK Investigational Site
Leiden, 2333 ZA
GSK Investigational Site
Nijmegen, 6525 GC
United Kingdom2 sites
GSK Investigational Site
London, WC1N 3JH
GSK Investigational Site
Newcastle upon Tyne, NE1 4LP
Argentina1 site
GSK Investigational Site
Ciudad Autónoma de Buenos Aires, Buenos Aires, C1125ABD
About this study
Official title: 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.
- Conditions
- Muscular Dystrophies
- Being tested
- GSK2402968
- Keywords
- 968, Duchenne, DMD, drisapersen
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GSK2402968Drug
6mg/kg/week
| Study group | Role | What they receive |
|---|---|---|
| Continuous Dosing | Experimental | GSK2402968 6mg/kg/week Receives: Drug: GSK2402968 |
| Intermittent Dosing | Experimental | GSK2402968 6mg/kg/week Receives: Drug: GSK2402968 |
| Natural History Observation | No intervention | The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Differences between the 6MWD at baseline and Week 104
Measured: 104 weeks
Secondary outcomes (11)
Timed Function tests
Measured: 104 weeks
Muscle strength
Measured: 104 weeks
North Star Ambulatory Assessment Scores
Measured: 104 weeks
Creatine kinase Serum concentrations
Measured: 104 weeks
Pulmonary Function
Measured: 104 weeks
Pediatric Quality of Life Neuromuscular module
Measured: 104weeks
Clinician Global Impression of Improvement
Measured: 104 weeks
Health Utilities Index
Measured: 104 weeks
Frequency of accidental falls during 6 Minute Walk Distance test
Measured: 104 weeks
Functional Outcomes Assessment
Measured: 104 weeks
Time to major disease milestones
Measured: 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2011 (estimated)
- Study startSep 2011
- Primary completionMar 2014
- Study completionMar 2014
Why it stopped: The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 233 participants (actual)
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- GSK Clinical Trials, Study director, GlaxoSmithKline
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01480245
- Sponsor's protocol ID
- 114349
- Status last verified
- Mar 2017
- First submitted
- 23 Nov 2011
- Last updated
- 23 Mar 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01480245
- Acronym / sponsor ID
- 114349
- Conditions
- Muscular Dystrophies
- Keywords
- 968, Duchenne, DMD, drisapersen
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.
- Enrolment
- 233 participants (actual)
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- Study start
- Sep 2011
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 28 Nov 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Continuous Dosing | Experimental | Drug: GSK2402968GSK2402968 6mg/kg/week |
| Intermittent Dosing | Experimental | Drug: GSK2402968GSK2402968 6mg/kg/week |
| Natural History Observation | No intervention | –The objective of this arm will be to explore DMD disease progression in a naturalistic setting once discontinuing active treatment |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GSK24029686mg/kg/week | Drug | Continuous Dosing, Intermittent Dosing |
Primary outcome
Differences between the 6MWD at baseline and Week 104
Time frame: 104 weeks
11 secondary outcomes
Timed Function tests
Time frame: 104 weeks
Muscle strength
Time frame: 104 weeks
North Star Ambulatory Assessment Scores
Time frame: 104 weeks
Creatine kinase Serum concentrations
Time frame: 104 weeks
Pulmonary Function
Time frame: 104 weeks
Pediatric Quality of Life Neuromuscular module
Time frame: 104weeks
Clinician Global Impression of Improvement
Time frame: 104 weeks
Health Utilities Index
Time frame: 104 weeks
Frequency of accidental falls during 6 Minute Walk Distance test
Time frame: 104 weeks
Functional Outcomes Assessment
Time frame: 104 weeks
Time to major disease milestones
Time frame: 104 weeks
Results and safety
The study was stopped early (reason given: The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
GSK Clinical Trials study director
GlaxoSmithKline
59 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing GSK2402968 in people with Muscular Dystrophies, run by GlaxoSmithKline at 59 sites in 24 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 59 sites in France, Brazil, Germany, Italy, Japan and 19 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The study was formally terminated given that GSK is not submitting an application for regulatory approval for drisapersen in Duchenne Muscular Dystrophy.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Differences between the 6MWD at baseline and Week 104 (time frame: 104 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2017.