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

Primary Purpose

Muscular Dystrophies

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
GSK2402968
matched placebo
Sponsored by
GlaxoSmithKline
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophies focused on measuring 968, muscular dystrophy, duchenne, DMD

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site
  • GSK Investigational Site

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Continuous regimen; 6mg/kg once weekly

Intermittent regimen; 6mg/kg twice weekly

Arm Description

Once 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

Outcomes

Primary Outcome Measures

To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD

Secondary Outcome Measures

To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects
To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD
To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD

Full Information

First Posted
June 29, 2010
Last Updated
August 21, 2014
Sponsor
GlaxoSmithKline
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1. Study Identification

Unique Protocol Identification Number
NCT01153932
Brief Title
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2013
Overall Recruitment Status
Completed
Study Start Date
September 2010 (undefined)
Primary Completion Date
March 2012 (Actual)
Study Completion Date
September 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
GlaxoSmithKline

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophies
Keywords
968, muscular dystrophy, duchenne, DMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
53 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Continuous regimen; 6mg/kg once weekly
Arm Type
Experimental
Arm Description
Once Weekly
Arm Title
Intermittent regimen; 6mg/kg twice weekly
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
GSK2402968
Other Intervention Name(s)
968
Intervention Description
Subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
matched placebo
Other Intervention Name(s)
Placebo
Intervention Description
Subcutaneous injection
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
GSK Clinical Trials
Organizational Affiliation
GlaxoSmithKline
Official's Role
Study Director
Facility Information:
Facility Name
GSK Investigational Site
City
WEstmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
GSK Investigational Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
GSK Investigational Site
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
GSK Investigational Site
City
Paris cedex 13
ZIP/Postal Code
75651
Country
France
Facility Name
GSK Investigational Site
City
Freiburg
State/Province
Baden-Wuerttemberg
ZIP/Postal Code
79106
Country
Germany
Facility Name
GSK Investigational Site
City
Essen
State/Province
Nordrhein-Westfalen
ZIP/Postal Code
45122
Country
Germany
Facility Name
GSK Investigational Site
City
Jerusalem
ZIP/Postal Code
91240
Country
Israel
Facility Name
GSK Investigational Site
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Facility Name
GSK Investigational Site
City
Esplugues (Barcelona)
ZIP/Postal Code
08950
Country
Spain
Facility Name
GSK Investigational Site
City
Valencia
ZIP/Postal Code
46009
Country
Spain
Facility Name
GSK Investigational Site
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
GSK Investigational Site
City
London
ZIP/Postal Code
WC1N 1EH
Country
United Kingdom
Facility Name
GSK Investigational Site
City
Newcastle upon Tyne
ZIP/Postal Code
NE1 3BZ
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
25209738
Citation
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.
Results Reference
derived

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

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