Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01153932
First received: June 29, 2010
Last updated: August 15, 2013
Last verified: August 2013
  Purpose

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.


Condition Intervention Phase
Muscular Dystrophies
Drug: GSK2402968
Drug: matched placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects [ Time Frame: one year ] [ Designated as safety issue: No ]
  • To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD [ Time Frame: one year ] [ Designated as safety issue: No ]

Enrollment: 53
Study Start Date: September 2010
Study Completion Date: September 2012
Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Continuous regimen; 6mg/kg once weekly
Once Weekly
Drug: GSK2402968
Subcutaneous injection
Other Name: 968
Drug: matched placebo
Subcutaneous injection
Other Name: Placebo
Experimental: Intermittent regimen; 6mg/kg twice weekly
Twice 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
Drug: GSK2402968
Subcutaneous injection
Other Name: 968
Drug: matched placebo
Subcutaneous injection
Other Name: Placebo

Detailed Description:

This is a phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study in ambulant subjects with DMD resulting from a mutation that can be corrected by exon skipping induced by GSK2402968. The study aims to randomise 54 subjects. There will be 2 parallel cohorts. Each cohort will include subjects on GSK2402968 and matched placebo in a 2:1 ratio.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01153932

Locations
Australia, New South Wales
GSK Investigational Site
WEstmead, New South Wales, Australia, 2145
Australia, Victoria
GSK Investigational Site
Parkville, Victoria, Australia, 3052
Belgium
GSK Investigational Site
Gent, Belgium, 9000
France
GSK Investigational Site
Paris cedex 13, France, 75651
Germany
GSK Investigational Site
Freiburg, Baden-Wuerttemberg, Germany, 79106
GSK Investigational Site
Essen, Nordrhein-Westfalen, Germany, 45122
Israel
GSK Investigational Site
Jerusalem, Israel, 91240
Netherlands
GSK Investigational Site
Nijmegen, Netherlands, 6525 GA
Spain
GSK Investigational Site
Esplugues (Barcelona), Spain, 08950
GSK Investigational Site
Valencia, Spain, 46009
Turkey
GSK Investigational Site
Ankara, Turkey, 06100
United Kingdom
GSK Investigational Site
London, United Kingdom, WC1N 1EH
GSK Investigational Site
Newcastle upon Tyne, United Kingdom, NE1 3BZ
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
  More Information

No publications provided

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01153932     History of Changes
Other Study ID Numbers: 114117
Study First Received: June 29, 2010
Last Updated: August 15, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
United Kingdom: Ethics Committee
France: Agence Française de Sécurité Sanitaire des Produits de Santé

Keywords provided by GlaxoSmithKline:
968
muscular dystrophy
duchenne
DMD

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 20, 2014