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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01153932
• Date of registration: 29/06/2010
• Prospective Registration: Yes
• Primary sponsor: GlaxoSmithKline
• Public title: Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
• Scientific 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
• Date of first enrolment: September 2010
• Target sample size: 53
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01153932
• 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
• Phase: Phase 2

Countries of recruitment

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

Contacts

• Name: GSK Clinical Trials
• Affiliation: GlaxoSmithKline

Key inclusion & exclusion 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



Age minimum: 5 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophies

Intervention(s)

Drug: matched placebo

Drug: GSK2402968

Primary Outcome(s)

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(s)

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]

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]

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]

Secondary ID(s)

114117

Source(s) of Monetary Support

Please refer to primary and secondary sponsors