Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
- Achondroplasia
At a glance
- Phase
- Phase 2
- Study type
- Interventional
- Sponsor
- BioMarin Pharmaceutical
- Enrolment target
- 160
- Started
- 20 April 2026
- Main results due
- June 2029
- Study sites
- 28
- Registry updated
- 24 September 2026
Can you take part?
- Ages 2 years to 17 years.
- Open to any sex.
- You need the condition being studied — healthy volunteers are not accepted.
These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.
Read the full eligibility criteria
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 with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \< 10 g/dL, vitamin D deficiency, significant hip pathology. 3. Have history of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension. 4. Have had bone fractures of the long bones or spine within 6 months prior to screening. 5. Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time 6. Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start
What this study is about
In the sponsor’s own words, from the registry.
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.
Study sites(28)
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Recruiting
Nemours Children's Health
Wilmington, Delaware, United States
Not yet recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Recruiting
Johns Hopkins Medicine
Baltimore, Maryland, United States
Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Recruiting
Texas Children's Hospital
Dallas, Texas, United States
Recruiting
Texas Children Hospital, Baylor College of Medicine
Houston, Texas, United States
Recruiting
Consano Clinical Research, LLC
San Antonio, Texas, United States
Recruiting
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Recruiting
McGill University Health Centre
Québec, Monteral, Canada
Not yet recruiting
Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Recruiting
Irccs Ospedale Gaslini Di Genova
Genova, Italy
Not yet recruiting
Fondazione IRCCS Ca Grande Ospedale Maggiore
Milan, Italy
Not yet recruiting
Hokkaido University Hospital
Hokkaido, Japan
Recruiting
Osaka City General Hospital
Osaka, Japan
Recruiting
National Center for Child Health and Development
Tokyo, Japan
Recruiting
Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego we Wroclawiu Klinika Pediatrii i Chorob Infekcyjnych
Wroclaw, Poland
Recruiting
Institutul National de Endocrinologie C.I.Parhon
Bucharest, Romania
Not yet recruiting
Craiova Emergency Clinical County
Craiova, Romania
Recruiting
Seoul National University Hospital
Seoul, South Korea
Recruiting
Pusan National University Yangsan Hospital
Yangsan, South Korea
Not yet recruiting
University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol, United Kingdom
Recruiting
Queen Elizabeth University Hospital
Glasgow, United Kingdom
Recruiting
Alder Hey Childrens NHS Foundation Trust
Liverpool, United Kingdom
Recruiting
Great Ormond Street Hospital
London, United Kingdom
Not yet recruiting
St Thomas Hospital
London, United Kingdom
Not yet recruiting
University NHS Foundation Trust NIHR Wellcome Trust Clinical Research Facility
Manchester, United Kingdom
Recruiting
Showing 28 of 28 sites. 20 of the 28 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.
Source: ClinicalTrials.gov record NCT07441876. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.
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