Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT07441876 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 160
Last updated 2026-05-07
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.
Conditions
Interventions
- DRUG
-
BMN 333
Administration: Weekly subcutaneous injection
- DRUG
-
Vosoritide Injection [Voxzogo]
Administration: Daily subcutaneous injection
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Medical Director, PhD · BioMarin Pharmaceutical
Study Design
- Allocation
- RANDOMIZED
- Purpose
- TREATMENT
- Masking
- SINGLE
- Model
- PARALLEL
Eligibility
- Min Age
- 2 Years
- Max Age
- 17 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-04-30
- Primary Completion
- 2029-06-30
- Completion
- 2029-09-30
- FDA Drug
- Yes
Countries
- United States
- Australia
Study Locations
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