A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
BioMarin Pharmaceutical
Summary
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.
Description
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 duratio…
Eligibility
- Age range
- 2–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent 2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses 3. Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3) 4. Are ambulatory and able to stand without assistance Exclusion Criteria: 1. Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency) 2. Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated…
Interventions
- DrugBMN 333
Administration: Weekly subcutaneous injection
- DrugVosoritide Injection [Voxzogo]
Administration: Daily subcutaneous injection
Locations (4)
- Consano Clinical Research, LLCCalifornia City, California
- UCSF Benioff Children's Hospital OaklandOakland, California
- Texas Children Hospital, Baylor College of Medicine, Houston TXHouston, Texas
- Murdoch Children's Research InstituteParkville, Victoria