Hypochondroplasia
Conditions
Brief summary
• Incidence of TEAEs versus placebo over the course of the study, • Incidence of SAEs versus placebo over the course of the study, • Changes in standard clinical laboratory values (urinalysis, chemistry, hematology) versus placebo over the course of the study, • Changes in vital signs versus placebo over the course of the study, • Change from baseline at Week 52 versus placebo in height Z-score
Detailed description
Change (Chg) baseline (bl) – Week (W)52 vs placebo (plcb) in height, 6-month interval AGV at W52 vs plcb, Chg bl - W52 vs plcb in upper to lower body segment ratio, Chg bl - W52 vs plcb in arm span, Chg bl - W52 vs plcb in total body BMD Zscore by DXA, Chg bl - W52 vs plcb in lumbar spine BMD Zscore by DXA, Chg bl - W52 vs plcb in tot. body BMC by DXA, Chg bl - W52 vs plcb in lumbar spine BMC by DXA, PK, Chg from pre-dose vs plcb in cGMP, Incidence of otitis media vs plcb, Seizure freq. vs plcb
Interventions
Sponsors
Biomarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Change (Chg) baseline (bl) – Week (W)52 vs placebo (plcb) in height, 6-month interval AGV at W52 vs plcb, Chg bl - W52 vs plcb in upper to lower body segment ratio, Chg bl - W52 vs plcb in arm span, Chg bl - W52 vs plcb in total body BMD Zscore by DXA, Chg bl - W52 vs plcb in lumbar spine BMD Zscore by DXA, Chg bl - W52 vs plcb in tot. body BMC by DXA, Chg bl - W52 vs plcb in lumbar spine BMC by DXA, PK, Chg from pre-dose vs plcb in cGMP, Incidence of otitis media vs plcb, Seizure freq. vs plcb | — |
Primary
| Measure | Time frame |
|---|---|
| • Incidence of TEAEs versus placebo over the course of the study, • Incidence of SAEs versus placebo over the course of the study, • Changes in standard clinical laboratory values (urinalysis, chemistry, hematology) versus placebo over the course of the study, • Changes in vital signs versus placebo over the course of the study, • Change from baseline at Week 52 versus placebo in height Z-score | — |
Countries
France, Germany, Italy
Outcome results
None listed