Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: GSK2402968
Product Code: GSK2402968
Pharmaceutical Form: Injection
Current Sponsor code: GSK2402968
Concentration unit: mg
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. Subjects who have successfully completed either the DMD114117 study or DMD114044 study, OR Subjects who withdrew due to safety/tolerability issues, which have since resolved and where in the opinion of the PI along with consultation of the Medical Monitor, it is considered that the benefit of further treatment with GSK2402968 outweighs the risk to the individual subject. OR Subjects who either complete or withdraw early from studies DMD114117 or DMD114044, who do not wish to continue treatment with GSK2402968 but who are willing to participate in the natural history observation arm. 2. Continued use of glucocorticoids with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to or cessation of glucocorticosteroids will be at the discretion of the PI in consultation with the subject/parent and the Medical Monitor. 3. Willing and able to comply with all protocol requirements and procedures, 4. Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations). 5. French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=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. Subject had a serious adverse experience or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in the opinion of the investigator could have been attributable to study medication, and which is ongoing at Visit 2. Once resolved, subject may be eligible to enrol following PI consultation with the Medical Monitor. 2. Use of anticoagulants, antithrombotics or antiplatelet agents, or previous treatment with investigational drugs except for GSK2402968, within 1 month of the first administration of study medication. 3. Current or anticipated participation in any other investigational clinical studies 4. History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current or history of renal or liver disease/impairment, history of inflammatory illness.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long term safety, tolerability and efficacy of subcutaneous 6mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044.; Secondary Objective: •To evaluate the long-term PK of subcutaneous 6 mg/kg/week GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044. •To evaluate the long-term impact on health-related quality of life (HRQoL) and functional outcomes of continued treatment with GSK2402968 in subjects with DMD who have participated in either DMD114117 or DMD114044. •To evaluate DMD disease progression and outcomes (clinical, HRQoL and functional) in subjects who discontinue active treatment during the conduct of study (natural history component). •To evaluate the long-term safety, efficacy and PK of an intermittent dosing option in those subjects unable to tolerate GSK2402968 6mg/kg/week dosing. ; Primary end point(s): Primary efficacy endpoint: • Muscle function using 6 minute walking distance (6MWD) test. ;Timepoint(s) of evaluation of this end point: Weeks 12,24,48,72 and Week 104 or Early withdrawal | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints: • Timed function tests (times and grading): • Rise from floor • 4 stair climb • 10m walk/run • Muscle strength (total score): knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors (as determined by handheld myometery) • North Star Ambulatory Assessment • Creatine kinase serum concentrations • Pulmonary function (FEV1, FVC, PCF, PF, sniff pressure test) • Pediatric Quality of Life Neuromuscular module (PedsQL) • Clinician Global Impression of Improvement (CGI-I) • Health Utilities Index (HUI) • Time to major disease milestones (e.g. loss of ambulation, night time ventilation) • Frequency of accidental falls (during 6MWD) • Functional Outcomes assessment Dystrophin expression NOTE: A muscle biopsy will be performed only if a case of an unexpected decrease in efficacy defined as: - 2 consecutive 20% decreases in 6MWD, after Week 24, in those subjects previously demonstrating improvement or maintenance of 6MWD (unless an alternative explanation can be provided by the investigator e.g. fall), or - a deterioration in efficacy based on any efficacy parameter which is worse than would be expected based on the natural history of the individual subject, in the opinion of the Investigator Safety endpoints: • Incidence and severity of adverse events. • Vital signs • ECG parameters • Safety haematology and biochemistry parameters including non-standard parameters such as coagulation parameters (in particular aPTT), serum cystatin C, Complement Factor C3, haptoglobin, fibrinogen, hsCR | — |
Countries
Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Czech Republic, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Korea, Republic of, Netherlands, Norway, Poland, Russian Federation, Spain, Taiwan, Turkey, United Kingdom
Contacts
Public ContactClinical Trials Helpdesk
GlaxoSmithKline
Outcome results
None listed