DRUG
BMN 333
Administration: Weekly subcutaneous injection
Status
Recruiting
Phase
Phase 2 / Phase 3
Enrollment
160
Locations
19
Results
Not posted
Publications
0
Study 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.
The main purpose of this study is to evaluate the effects of BMN 333 on growth compared with vosoritide in participants with achondroplasia who have not received any growth-promoting treatments. The study includes 2 parts: the Phase 2 part will select the optimal BMN 333 dose to be used in Phase 3 and determine study continuation into Phase 3; the Phase 3 part will compare the effects of the selected dose of BMN 333 with vosoritide. Study details for either Phase 2 or Phase 3 include the following: * Study duration: up to 61 weeks (from screening to Safety Follow-up visit) * Treatment duration: 52 weeks. Treatment frequency: BMN 333, once weekly; vosoritide, once daily
Interventions
DRUG
Administration: Weekly subcutaneous injection
DRUG
Administration: Daily subcutaneous injection
Timeline
First posted
Mar 2, 2026
Study start
Apr 20, 2026
Primary completion
Jun 2029
Study completion
Sep 2029
Results posted
Not reported
Registry updated
Jul 24, 2026
Outcomes
Phase 2: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data]
Time frame · 52 weeks
Phase 3: Annualized Growth Velocity (AGV) at Week 52
Time frame · 52 weeks
Phase 2: AGV at Weeks 26 and 52
Time frame · 26 and 52 weeks
Phase 2: Change from Baseline in standing height
Time frame · 26 and 52 weeks
Measured in centimeters
Phase 2: Change from Baseline in height Z-score
Time frame · 26 and 52 weeks
Phase 2: Change from Baseline in upper to lower body segment ratio
Time frame · 26 and 52 weeks
Phase 2: Incidence of adverse events (AEs)
Time frame · 52 weeks
Phase 2: Incidence of serious adverse events (SAEs)
Time frame · 52 weeks
Phase 2: Incidence of events of interest (EOIs)
Time frame · 52 weeks
Phase 2: Maximum concentration (Cmax) of BMN 333 in plasma
Time frame · 52 weeks
Phase 2: Maximum concentration (Cmax) of released vosoritide in plasma
Time frame · 52 weeks
Phase 2: Time to reach maximum concentration (Tmax) for BMN 333
Time frame · 52 weeks
Eligibility
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
Study locations
Australia · Canada · Italy · Japan · Poland · Romania · South Korea · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Nemours Children's Health
Wilmington, Delaware, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins Medicine
Baltimore, Maryland, United States
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
Texas Children Hospital, Baylor College of Medicine
Houston, Texas, United States
Consano Clinical Research, LLC
San Antonio, Texas, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Irccs Ospedale Gaslini Di Genova
Genova, Italy
Osaka City General Hospital
Osaka, Japan
Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego we Wroclawiu Klinika Pediatrii i Chorob Infekcyjnych
Wroclaw, Poland
Institutul National de Endocrinologie C.I.Parhon
Bucharest, Romania
Craiova Emergency Clinical County
Craiova, Romania
Seoul National University Hospital
Seoul, South Korea
Pusan National University Yangsan Hospital
Yangsan, South Korea
University Hospitals Bristol NHS Foundation Trust - Bristol Royal Hospital for Children
Bristol, United Kingdom
Publications
No PMID-linked publications were present in this registry snapshot.
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