Endpoints
Primary endpoints
- Pharmacokinetics AUCτ,ss (Weeks 2 & 26)
- Treatment-emergent adverse events (Week 24)
Secondary endpoints
- Pathologic findings of epiphyseal growth plate (Weeks 24 & 52)
- Pathologic findings on dental examination or imaging (Weeks 24 & 52)
- Treatment-emergent adverse events (whole trial)
- Change in height, sitting height and leg length from baseline (Weeks 24, 52, 76 & 100)
- FVC % predicted change from baseline (Weeks 24 & 52)
- Oxygen saturation in air change from baseline (Weeks 24 & 52)
- Pediatric Quality of Life Questionnaire (PedsQL™) (Weeks 24 & 52)
- 6-minute walk distance change from baseline (Weeks 24 & 52)
- Additional efficacy and safety endpoints (over longer term)