Actively Recruiting
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Led by BioMarin Pharmaceutical · Updated on 2026-05-07
160
Participants Needed
4
Research Sites
178 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be aged 2 to less than 11 years (Phase 2) or 2 to less than 18 years (Phase 3) at consent
- Participants must have achondroplasia confirmed by genetic testing and open growth plates
- Participants are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
- Participants must be able to walk and stand without assistance
You will not qualify if you...
- Participants with any short stature condition other than achondroplasia (e.g., hypochondroplasia, trisomy 21, pseudoachondroplasia, growth hormone deficiency)
- Participants with untreated thyroid disorders, poorly controlled diabetes, autoimmune or inflammatory diseases, autonomic neuropathy, anemia (hemoglobin below 10 g/dL), vitamin D deficiency, or significant hip problems
- Participants with a history of kidney or heart disease that increases cardiac risk during low blood pressure
- Participants with bone fractures in long bones or spine within 6 months before screening
- Participants who have used vosoritide, other approved or investigational products or devices for achondroplasia or short stature
- Participants treated with growth hormone, insulin-like growth factor 1, or anabolic steroids within 6 months before treatment start
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Consano Clinical Research, LLC
California City, California, United States, 78231
Actively Recruiting
2
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
3
Texas Children Hospital, Baylor College of Medicine, Houston TX
Houston, Texas, United States, 77030
Actively Recruiting
4
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
Research Team
T
Trial Specialist
CONTACT
S
Study Manager
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
6
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