This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.
The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following: * Study duration: up to 61 weeks (from screening to Safety Follow-up visit) * Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
160
Administration: Weekly subcutaneous injection
Administration: Daily subcutaneous injection
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
RECRUITINGTexas Children Hospital, Baylor College of Medicine, Houston TX
Houston, Texas, United States
RECRUITINGPhase 2: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data]
Time frame: 52 weeks
Phase 3: Annualized Growth Velocity (AGV) at Week 52
Time frame: 52 weeks
Phase 2: AGV at Weeks 26 and 52
Time frame: 26 and 52 weeks
Phase 2: Change from Baseline in standing height
Measured in centimeters
Time frame: 26 and 52 weeks
Phase 2: Change from Baseline in height Z-score
Time frame: 26 and 52 weeks
Phase 2: Change from Baseline in upper to lower body segment ratio
Time frame: 26 and 52 weeks
Phase 2: Incidence of adverse events (AEs)
Time frame: 52 weeks
Phase 2: Incidence of serious adverse events (SAEs)
Time frame: 52 weeks
Phase 2: Incidence of events of interest (EOIs)
Time frame: 52 weeks
Phase 2: Maximum concentration (Cmax) of BMN 333 in plasma
Time frame: 52 weeks
Phase 2: Maximum concentration (Cmax) of released vosoritide in plasma
Time frame: 52 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Phase 2: Time to reach maximum concentration (Tmax) for BMN 333
Time frame: 52 weeks
Phase 2: Time to reach maximum concentration (Tmax) for released vosoritide
Time frame: 52 weeks
Phase 2: Lowest concentration (C trough) of BMN 333 in plasma
Time frame: 52 weeks
Phase 2: Lowest concentration (C trough) of released vosoritide in plasma
Time frame: 52 weeks
Phase 3: Change from Baseline in standing height
Measured in centimeters
Time frame: 52 weeks
Phase 3: Change from Baseline in height Z-score
Time frame: 52 weeks
Phase 3: Change from Baseline in upper to lower body segment ratio
Time frame: 52 weeks