Open label study of GSK2402968 in subjects with Duchenne Muscular Dystrophy
Trial overview
Change from Baseline in muscle function using the 6 minute walking distance (6MWD) test assessed at Week 104
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in muscle strength measured in pounds as total as well as individual knee flexors, knee extensors, elbow flexors, elbow extensors, shoulder abductors and hip flexors
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in the following timed function tests (times and grading)-rise from floor
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in the following timed function tests (times and grading)-4 stair climb ascent and descent
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in the following timed function tests (times and grading)-10 meter walk/run
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in the North Star Ambulatory Assessment (NSAA) total score
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in creatine kinase (CK) serum concentrations
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104
Change from Baseline in lung function test: forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1)
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in lung function test: peak cough flow (PCF) and peak flow (PF)
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in lung function test: sniff test
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in the Pediatric Quality of Life Neuromuscular module (PedsQL) total score
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in the Pediatric Quality of Life Neuromuscular module (PedsQL) subscale score
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with Clinician Global Impression of Improvement (CGI-I) till Week 104
Timeframe: Baseline (Week 0) and Week 24, 48, 72, 104, follow-up: 12 week post-dose
Change from Baseline in Health Utilities Index Scores (HUI)
Timeframe: Baseline (Week 0) and Week 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with accidental falls (during 6MWD)
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with functional outcomes assessment survey individual scores-general health-Child's health
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with functional outcomes assessment survey individual scores-general health -Child's condition
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with functional outcomes assessment survey individual scores-mobility (ability to move around)
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with functional outcomes assessment survey individual scores-physical activities
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with functional outcomes assessment survey individual scores-hand dexterity coordination
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with functional outcomes assessment survey individual scores-use of assistive devices (example: orthotics, wheelchair)
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Number of participants with major disease milestones (example loss of ambulation)
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Muscle biopsy - dystrophin expression
Timeframe: Baseline (Week 0) and Week 12, 24, 48, 72, 104, follow-up: 12 week post-dose
Participation criteria
- Previous participation in either DMD114117 or DMD114044
- Continued use of glucocorticoids
- Subject experienced a serious adverse event or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in opinion of the investigator could have been attributable to study medication and is ongoing,
- Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs,except GSK2402968, within 1 month of the first administration of study medication,
- Previous participation in either DMD114117 or DMD114044
- Continued use of glucocorticoids
- Willing and able to comply with all protocol requirements
- Able to give informed consent
- French subjects: Eligible for inclusion only if either affiliated to or a beneficiary of a social security category.
- Subject experienced a serious adverse event or who met safety stopping criteria that remains unresolved from DMD114117 or DMD114044, which in opinion of the investigator could have been attributable to study medication and is ongoing,
- Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs,except GSK2402968, within 1 month of the first administration of study medication,
- Current or anticipated participation in any investigational clinical studies,
- History of significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. current history of renal or liver disease/impairment, history of inflammatory disease.
Trial location(s)
Study documents
No study documents available.
Results overview
Results posted on ClinicalTrials.gov
Plain language summaries
Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.