Achondroplasia
Conditions
Brief summary
1)Key efficacy endpoints: a)Change from baseline in AGV b) Change from baseline in height Z-score c)Change from baseline in upper to lower body segment ratio, 2)Safety assessments: Incidence, severity, and relationship to study drug of all treatment-emergent adverse events (TEAEs)., 3)Procedures/ interventions/ surgeries, imaging assessments, clinical laboratory assessments, Child Behavior Checklist (CBCL), vital signs, electrocardiogram (ECG), and hip clinical assessment.
Detailed description
Change from baseline in: 1)Upper Arm Length to Lower Arm (Forearm), Length Ratio, 2)Upper Leg Length (Thigh) to Knee to Heel Length Ratio, 3)Upper Leg Length (Thigh) to Tibial Length Ratio, 4)Arm Span to Standing Height Ratio, 5)Bone age, bone age Z-score, bone mineral density, (BMD), BMD Z-score, and bone mineral content (BMC), 6)QoLISSY and PedsQL domain and total scores
Interventions
Sponsors
Biomarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1)Key efficacy endpoints: a)Change from baseline in AGV b) Change from baseline in height Z-score c)Change from baseline in upper to lower body segment ratio, 2)Safety assessments: Incidence, severity, and relationship to study drug of all treatment-emergent adverse events (TEAEs)., 3)Procedures/ interventions/ surgeries, imaging assessments, clinical laboratory assessments, Child Behavior Checklist (CBCL), vital signs, electrocardiogram (ECG), and hip clinical assessment. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in: 1)Upper Arm Length to Lower Arm (Forearm), Length Ratio, 2)Upper Leg Length (Thigh) to Knee to Heel Length Ratio, 3)Upper Leg Length (Thigh) to Tibial Length Ratio, 4)Arm Span to Standing Height Ratio, 5)Bone age, bone age Z-score, bone mineral density, (BMD), BMD Z-score, and bone mineral content (BMC), 6)QoLISSY and PedsQL domain and total scores | — |
Countries
Germany, Spain
Outcome results
None listed