Duchenne Muscular Dystrophy MedDRA version: 14.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
GlaxoSmithKline Research and Development LTD
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. 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), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by GSK2402968-induced DMD exon 51 skipping, 2. Aged at least 5 years, 3. Male, 4. Life expectancy of at least 1 year, 5. Able to rise from floor in =7 seconds (without aids/orthoses), 6. Able to complete the 6MWD test with a distance of at least 75m. In addition, results of 6MWD must be within 20% of each other at each pre-drug visit, 7. 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, 8. 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 missing exon for DMD that cannot be treated with GSK2402968, 2. Current or history of liver or renal disease or impairment, 3. Acute illness within 4 weeks of the first anticipated administration of study medication which may interfere with study assessments, 4. 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 the first administration of study medication, 5. Current or anticipated participation in any investigational clinical studies, 6. Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening, 7. Symptomatic cardiomyopathy. If subject has a left ventricular ejection fraction <45% at Screening, the investigator should discuss inclusion of subject in the study with the medical monitor 8. Children in Care. The definition of a Child in Care is a child who has been placed under the control or protection of an agency, organisation, 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 efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD.;Secondary Objective: • To assess the safety and tolerability of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD. • To assess the PK of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD. • To assess long term efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 48 weeks in ambulant subjects with DMD. ;Primary end point(s): Primary efficacy endpoint: • Muscle function using 6 minute walking distance (6MWD) test. ;Timepoint(s) of evaluation of this end point: Every 12 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Timed function tests (times and grading): -rise from floor -10m walk/run -4-stair climb • Muscle strength (total score): knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors (as determined by handheld dynamometry) • North Star Ambulatory Assessment • Frequency of accidental falls (during 6MWD) • Time to loss of ambulation • Creatine kinase serum concentrations • Pulmonary function (FEV1, FVC, MIP, MEP, PCF, PF) • Dystrophin expression (muscle biopsies) ;Timepoint(s) of evaluation of this end point: Various | — |
Countries
Australia, France, Germany, Israel, Netherlands, Spain, Turkey, United Kingdom
Contacts
Public ContactClinical Trials Helpdesk
GlaxoSmithKline Research & Development Ltd
Outcome results
None listed