Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
A Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy
1 other identifier
interventional
53
9 countries
13
Brief Summary
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Sep 2010
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 29, 2010
CompletedFirst Posted
Study publicly available on registry
June 30, 2010
CompletedStudy Start
First participant enrolled
September 1, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2012
CompletedAugust 25, 2014
August 1, 2013
1.5 years
June 29, 2010
August 21, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD
48 weeks
Secondary Outcomes (3)
To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects
one year
To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD
48 weeks
To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD
one year
Study Arms (2)
Continuous regimen; 6mg/kg once weekly
EXPERIMENTALOnce Weekly
Intermittent regimen; 6mg/kg twice weekly
EXPERIMENTALTwice weekly on 1st, 3rd and 5th weeks, once weekly on 2nd, 4th and 6th weeks, and no active drug on 7th to 10th week of each 10 week cycle
Interventions
Subcutaneous injection
Subcutaneous injection
Eligibility Criteria
You may qualify if:
- Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a state-of-the-art DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by GSK2402968-induced DMD exon 51 skipping,
- Males, at least 5 years of age and with a life expectancy of at least 1 year
- Able to rise from floor in ≤7 seconds (without aids/orthoses),
- Able to complete the 6MWD test with a distance of at least 75m
- Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study
- QTc \<450msec
- On adequate contraception
- Able to comply with and complete all protocol requirements
You may not qualify if:
- any additional missing exon for DMD
- Current of history of liver or renal disease or impairment
- Acute illness within 4 weeks of the first dose
- Use of prohibited meds within 6 months of fist dose
- Current participation in any other investigational clinical trial
- Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening
- Symptomatic cardiomyopathy
- Children in Care
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- GlaxoSmithKlinelead
Study Sites (13)
GSK Investigational Site
Westmead, New South Wales, 2145, Australia
GSK Investigational Site
Parkville, Victoria, 3052, Australia
GSK Investigational Site
Ghent, 9000, Belgium
GSK Investigational Site
Paris, 75651, France
GSK Investigational Site
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
GSK Investigational Site
Essen, North Rhine-Westphalia, 45122, Germany
GSK Investigational Site
Jerusalem, 91240, Israel
GSK Investigational Site
Nijmegen, 6525 GA, Netherlands
GSK Investigational Site
Esplugues (Barcelona), 08950, Spain
GSK Investigational Site
Valencia, 46009, Spain
GSK Investigational Site
Ankara, 06100, Turkey (Türkiye)
GSK Investigational Site
London, WC1N 1EH, United Kingdom
GSK Investigational Site
Newcastle upon Tyne, NE1 3BZ, United Kingdom
Related Publications (1)
Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, De Kimpe SJ, Eagle M, Guglieri M, Hood S, Liefaard L, Lourbakos A, Morgan A, Nakielny J, Quarcoo N, Ricotti V, Rolfe K, Servais L, Wardell C, Wilson R, Wright P, Kraus JE. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014 Oct;13(10):987-96. doi: 10.1016/S1474-4422(14)70195-4. Epub 2014 Sep 7.
PMID: 25209738DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
GSK Clinical Trials
GlaxoSmithKline
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 29, 2010
First Posted
June 30, 2010
Study Start
September 1, 2010
Primary Completion
March 1, 2012
Study Completion
September 1, 2012
Last Updated
August 25, 2014
Record last verified: 2013-08