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
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
NOT_YET_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.
Johns Hopkins Medicine Julie
Baltimore, Maryland, United States
NOT_YET_RECRUITINGTexas Children Hospital, Baylor College of Medicine, Houston TX
Houston, Texas, United States
RECRUITINGConsano Clinical Research, LLC
San Antonio, Texas, United States
RECRUITINGMurdoch Children's Research Institute
Parkville, Victoria, Australia
RECRUITINGIrccs Ospedale Gaslini Di Genova
Genova, Italy
NOT_YET_RECRUITINGCraiova Emergency Clinical County
Craiova, Romania
NOT_YET_RECRUITINGSeoul National University Hospital
Seoul, South Korea
NOT_YET_RECRUITINGUniversity Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol, United Kingdom
NOT_YET_RECRUITINGPhase 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