A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children with Hypochondroplasia, Aged 0 to < 36 Months

Condition studied

Hypochondroplasia

Investigational medicinal product(s)

Eligibility

Primary endpoint

• 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

Endpoint detail

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