Actively Recruiting
A Multicenter, Randomized Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Led by BioMarin Pharmaceutical · Updated on 2026-05-29
160
Participants Needed
10
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of BMN 333 compared to vosoritide in children with achondroplasia who have not previously received growth-promoting treatments. This multicenter, multinational, randomized Phase 2/3 study aims to find the best dose of BMN 333 in Phase 2 and then compare its effects with vosoritide in Phase 3. The study focuses on growth outcomes in pediatric participants aged 2 to under 18 years with achondroplasia. During the study, participants will receive either BMN 333 once weekly or vosoritide once daily through subcutaneous injections. The Phase 2 part includes multiple dose levels of BMN 333 to identify the optimal dose for Phase 3. The treatment period lasts 52 weeks, with total study participation up to 61 weeks including screening and safety follow-up. Participants will be randomized to different treatment groups during both phases. Participants will undergo assessments of growth velocity, standing height, height Z-score, and body segment ratios at several time points, including weeks 26, 39, and 52. Safety will be monitored through adverse event tracking and blood tests measuring drug levels. The main outcome is annualized growth velocity at week 52. The study also includes follow-up visits for safety monitoring after treatment ends, with total participation lasting up to 61 weeks.
CONDITIONS
Brief 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 aged 2 to under 11 years for Phase 2, or 2 to under 18 years for Phase 3, at the time of informed consent
- Confirmed diagnosis of achondroplasia by genetic testing with open growth plates
- Tanner Stage I for Phase 2 or any Tanner stage for Phase 3
- Able to walk and stand without assistance
You will not qualify if you...
- Presence of any short stature condition other than achondroplasia (e.g., hypochondroplasia, trisomy 21, pseudoachondroplasia, growth hormone deficiency)
- Disorders such as untreated thyroid disease, poorly controlled diabetes, autoimmune inflammatory diseases, inflammatory bowel disease, autonomic neuropathy, anemia with hemoglobin less than 10 g/dL, vitamin D deficiency, or significant hip problems
- History of kidney problems or heart/cardiovascular disease increasing risk of cardiac issues with low blood pressure
- Bone fractures in long bones or spine within 6 months before screening
- Previous use of vosoritide, other approved treatments (except growth hormone under certain conditions), investigational products, or devices for achondroplasia or short stature
- Treatment with growth hormone, insulin-like growth factor 1, or anabolic steroids within 6 months before starting treatment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 52 weeks
Participants receive either BMN 333 by weekly subcutaneous injection or vosoritide by daily subcutaneous injection to evaluate effects on growth in children with achondroplasia.
Weekly visits for BMN 333 group and daily dosing for vosoritide group
Duration - Up to 9 weeks
Participants are monitored for safety after completing treatment.
1 visit (in-person)
Trial Site Locations
Total: 10 locations
1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
2
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Not Yet Recruiting
3
Johns Hopkins Medicine Julie
Baltimore, Maryland, United States, 21205
Not Yet Recruiting
4
Texas Children Hospital, Baylor College of Medicine, Houston TX
Houston, Texas, United States, 77030
Actively Recruiting
5
Consano Clinical Research, LLC
San Antonio, Texas, United States, 78231
Actively Recruiting
6
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
7
Irccs Ospedale Gaslini Di Genova
Genova, Italy, 16147
Not Yet Recruiting
8
Craiova Emergency Clinical County
Craiova, Romania, 200642
Not Yet Recruiting
9
Seoul National University Hospital
Seoul, South Korea
Not Yet Recruiting
10
University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol, United Kingdom
Not Yet Recruiting
Research Team
T
Trial Specialist
S
Study Manager
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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