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A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
study id #: NCT01128855
condition: Muscular Dystrophies
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968, 12 mg/kg GSK2402968, Placebo
mechanism of action: Exon-skipping to promote dystrophin production
start date: July 12, 2010
estimated completion: October 25, 2011
phase of development: Phase 1
size / enrollment: 20
- Primary Pharmacokinetic Variables: AUC, Cmax,t-max, CL/F [ Time Frame: 35 days ]
- Incidence of Adverse Events [ Time Frame: 35 days ]
- Incidence of Injection Site Reactions [ Time Frame: 35 days ]
• Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK2402968.
• Age 9 years old or greater at Screening;
• Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
• Life expectancy at least three years;
• Willingness and ability to comply with all protocol requirements and procedures;
• QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread;
• Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug;
• Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
• Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968;
• Current or history of liver or renal disease;
• Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments;
• Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication;
• Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication;
• Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening;
• Symptomatic cardiomyopathy;
• Use of alcohol from Screening through to the 1 month Follow-up visit;
• Any Child in Care.
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