Achondroplasia in Children and Adolescents
Conditions
Brief summary
Incidence of treatment emergent adverse events (TEAEs) [Time Frame: Through trial completion], Height Z-scores [Time Frame: Through trial completion]
Detailed description
Annualized growth velocity (AGV) (cm/year), Near final adult height [Time Frame: Through trial completion], Safety assessments: • Laboratory results of safety blood parameters • Vital signs (BP, HR, body temperature) • Abnormal physical examination findings • 12-lead electrocardiogram (ECG) • Tanner stage • Imaging assessments of bone age and bone related safety events (e.g. fracture, skeletal deformities, joint damage, slipped capital femoral epiphysis, avascular necrosis and osteonecrosis) • Tolerability and acceptability [Time Frame: Through trial completion], Plasma concentrations of Total CNP, Free CNP, and mPEG [Time Frame: Through treatment completion], Detection and analysis of anti-drug antibodies (ADAs) [Time Frame: Through trial completion]
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment emergent adverse events (TEAEs) [Time Frame: Through trial completion], Height Z-scores [Time Frame: Through trial completion] | — |
Secondary
| Measure | Time frame |
|---|---|
| Annualized growth velocity (AGV) (cm/year), Near final adult height [Time Frame: Through trial completion], Safety assessments: • Laboratory results of safety blood parameters • Vital signs (BP, HR, body temperature) • Abnormal physical examination findings • 12-lead electrocardiogram (ECG) • Tanner stage • Imaging assessments of bone age and bone related safety events (e.g. fracture, skeletal deformities, joint damage, slipped capital femoral epiphysis, avascular necrosis and osteonecrosis) • Tolerability and acceptability [Time Frame: Through trial completion], Plasma concentrations of Total CNP, Free CNP, and mPEG [Time Frame: Through treatment completion], Detection and analysis of anti-drug antibodies (ADAs) [Time Frame: Through trial completion] | — |
Countries
Austria, Denmark, Germany, Ireland, Portugal, Spain