Achondroplasia
Conditions
Brief summary
Phase 2: Predicted AGV at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data]), Phase 3: AGV at Week 52
Detailed description
Phase 2: 1.AGV at Weeks 26 and 52 2.Change from Baseline in standing height, height Z-score and in upper to lower body segment ratio at Weeks 26 and 52 3.Incidence of AEs, SAEs, and EOIsa; physical examinations; vital signs; ECG findings; imaging results (X-ray and DXA); clinical laboratory tests., Phase 3: • Change from Baseline in standing height, height Z-score, upper to lower body segment ratio at Week 52
Interventions
Sponsors
Biomarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase 2: Predicted AGV at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data]), Phase 3: AGV at Week 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Phase 2: 1.AGV at Weeks 26 and 52 2.Change from Baseline in standing height, height Z-score and in upper to lower body segment ratio at Weeks 26 and 52 3.Incidence of AEs, SAEs, and EOIsa; physical examinations; vital signs; ECG findings; imaging results (X-ray and DXA); clinical laboratory tests., Phase 3: • Change from Baseline in standing height, height Z-score, upper to lower body segment ratio at Week 52 | — |
Outcome results
None listed