Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Recruiting now · Phase 2/Phase 3

Conditions studied: Achondroplasia

In brief

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.

Key facts

Study ID

NCT07441876

Run by

BioMarin Pharmaceutical

People needed

160

Starts

2026-04-20

Expected to finish

2029-09-01

Last updated by the study team

2026-07-24

Who can join

Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Participants must have ACH (confirmed by documented genetic testing) and open epiphyses
• Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
• Are ambulatory and able to stand without assistance

You may not qualify if…

• Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency)
• 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.
• 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.
• Have had bone fractures of the long bones or spine within 6 months prior to screening.
• 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
• Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start

Where it is running

• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (enrolling)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Johns Hopkins Medicine — Baltimore, Maryland, United States (enrolling)
• Texas Children Hospital, Baylor College of Medicine — Houston, Texas, United States (enrolling)
• Consano Clinical Research, LLC — San Antonio, Texas, United States (enrolling)
• Murdoch Children's Research Institute — Parkville, Victoria, Australia (enrolling)
• Osaka City General Hospital — Osaka, Japan (enrolling)
• Seoul National University Hospital — Seoul, South Korea (enrolling)
• Craiova Emergency Clinical County — Craiova, Romania
• University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children — Bristol, United Kingdom
• Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec, Canada
• Irccs Ospedale Gaslini Di Genova — Genova, Italy
• Nemours Children's Health — Wilmington, Delaware, United States
• Pusan National University Yangsan Hospital — Yangsan, South Korea
• Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego we Wroclawiu Klinika Pediatrii i Chorob Infekcyjnych — Wroclaw, Poland
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Texas Southwestern Medical Center — Dallas, Texas, United States
• Institutul National de Endocrinologie C.I.Parhon — Bucharest, Romania

Full record on ClinicalTrials.gov

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