Muscular Dystrophies
Conditions
Keywords
968, muscular dystrophy, duchenne, DMD
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.
Interventions
DRUGGSK2402968
Subcutaneous injection
DRUGmatched placebo
Subcutaneous injection
Sponsors
GlaxoSmithKline
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
5 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD | 48 weeks |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
Countries
Australia, Belgium, France, Germany, Israel, Netherlands, Spain, Turkey (Türkiye), United Kingdom
Outcome results
None listed