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Recruiting Phase 2/3

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

NCT07441876 · tracked via the Priya Life Science Italy tracker
Phase
Phase 2/3
Started
2026-04-20
Last updated
2026-09-24

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

BMN 333Vosoritide Injection [Voxzogo] →

BMN 333: Administration: Weekly subcutaneous injection

Vosoritide Injection [Voxzogo]: Administration: Daily subcutaneous injection

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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Phase 2: Predicted Annualized Growth Velocity (AGV) at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data] — 52 weeks
  • Phase 3: Annualized Growth Velocity (AGV) at Week 52 — 52 weeks

Trial sites (28)

FacilityCityRegionStatus
UCSF Benioff Children's Hospital Oakland Oakland California Recruiting
Nemours Children's Health Wilmington Delaware Not Yet Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
Johns Hopkins Medicine Baltimore Maryland Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Texas Children's Hospital Dallas Texas Recruiting
Texas Children Hospital, Baylor College of Medicine Houston Texas Recruiting
Consano Clinical Research, LLC San Antonio Texas Recruiting
Murdoch Children's Research Institute Parkville Victoria Recruiting
McGill University Health Centre Québec Monteral Not Yet Recruiting
Universite de Montreal - Centre Hospitalier Universitaire Sainte-Justine Montreal Quebec 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
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07441876 on ClinicalTrials.gov ↗ ← All trials in Italy