Duchenne Muscular Dystrophy MedDRA version: 12.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Conditions
Interventions
Product Name: GSK2402968
Product Code: GSK2402968
Pharmaceutical Form: Injection*
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentra
Sponsors
GlaxoSmithKline Research and Development LTD
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Subjects eligible for enrolment in the study must meet all of the following criteria: 1. 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. 2. Age 9 years old or greater at Screening; 3. Male; 4. Non-ambulant (at least 1 year in a wheelchair) within the last 4 years; 5. Life expectancy at least three years; 6. Willingness and ability to comply with all protocol requirements and procedures; 7. QTc =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects meeting any of the following criteria must not be enrolled in the study: 1. Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968; 2. Current or history of liver or renal disease; 3. Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments; 4. Daytime ventilator-dependency (except for daytime naps); 5. Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, within 6 months of the first administration of study medication; and idebenone or other forms of Coenzyme Q10 within 1 month of study medication. 6. Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication; 7. Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening; 8. Symptomatic cardiomyopathy; 9. Use of alcohol from Screening through to the 1 month Follow-up visit (Day 29-35, inclusive); 10. Any Child in Care. (The definition of a Child in Care is a child who has been placed under the control or protection of an agency, organization, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. The definition of a child in care can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or has an appointed legal guardian).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the PK, safety and tolerability of GSK2402968 after a single subcutaneous administration at different dose levels in non-ambulatory subjects with Duchenne muscular dystrophy.;Secondary Objective: No Secondary objectives;Primary end point(s): Primary Pharmacokinetic Variables: • AUC0-24h, AUC0-7d, AUC0-last • Cmax, • tmax • CL/F. Safety Variables: • Adverse events • Physical examination including local tolerability • Vital signs • 12-lead ECGs, • Safety hematology and biochemistry parameters including non-standard parameters such as coagulation parameters (in particular aPTT), cystatin C, haptoglobulin, fibrinogen, CRP, complement split products (C3a, SC5b-9, Bb), inflammation markers (IL-6, TNF-a and MCP-1) and antibodies to dystrophin. • Urinalysis: including: protein, creatinine, a1-microglobulin and protein/ creatinine ratio. | — |
Countries
France
Outcome results
None listed