A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 versus Vosoritide in Children with Achondroplasia

Condition studied

Achondroplasia

Investigational medicinal product(s)

Eligibility

Primary endpoint

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

Endpoint detail

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