Duchenne Muscular Dystrophy (DMD)
Conditions
Brief summary
- Vital signs - Physical examination - Clinical laboratory tests o Hematology and clinical chemistry o Urinalysis o Urine cytology - Antibodies to dystrophin and viltolarsen - 12-lead electrocardiogram (ECG) - Renal ultrasound - Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Detailed description
Secondary: - Time to Stand Test (TTSTAND) - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Stairs Test (TTCLIMB) - Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only)
Interventions
DRUGViltolarsen
Sponsors
Ns Pharma Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Vital signs - Physical examination - Clinical laboratory tests o Hematology and clinical chemistry o Urinalysis o Urine cytology - Antibodies to dystrophin and viltolarsen - 12-lead electrocardiogram (ECG) - Renal ultrasound - Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary: - Time to Stand Test (TTSTAND) - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Stairs Test (TTCLIMB) - Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only) | — |
Countries
Czechia, Greece, Italy, Netherlands, Norway, Spain
Outcome results
None listed