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

Muscular Dystrophies Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01153932
• Other study ID #: 114117
• Status: Completed
• Phase: Phase 2
• First received: June 29, 2010
• Last updated: August 21, 2014
• Start date: September 2010
• Est. completion date: September 2012

Study information

• Verified date: August 2013
• Source: GlaxoSmithKline
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 53
• Est. completion date: September 2012
• Est. primary completion date: March 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• GSK2402968
Subcutaneous injection
• matched placebo
Subcutaneous injection

Locations

Country	Name	City	State
Australia	GSK Investigational Site	Parkville	Victoria
Australia	GSK Investigational Site	WEstmead	New South Wales
Belgium	GSK Investigational Site	Gent
France	GSK Investigational Site	Paris cedex 13
Germany	GSK Investigational Site	Essen	Nordrhein-Westfalen
Germany	GSK Investigational Site	Freiburg	Baden-Wuerttemberg
Israel	GSK Investigational Site	Jerusalem
Netherlands	GSK Investigational Site	Nijmegen
Spain	GSK Investigational Site	Esplugues (Barcelona)
Spain	GSK Investigational Site	Valencia
Turkey	GSK Investigational Site	Ankara
United Kingdom	GSK Investigational Site	London
United Kingdom	GSK Investigational Site	Newcastle upon Tyne

Sponsors (1)

Lead Sponsor	Collaborator
GlaxoSmithKline

Countries where clinical trial is conducted

Australia,  Belgium,  France,  Germany,  Israel,  Netherlands,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD		48 weeks
Secondary	To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects		one year
Secondary	To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD		48 weeks
Secondary	To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD		one year