NCT07441876
Achondroplasia
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
2 Years to 17 Years
No
BMN 333, Vosoritide Injection [Voxzogo]
Phase 2/Phase 3
Interventional
160
2026-04-20
2026-08-21
Oakland, California, United States
Trial Status Recruiting
Hind Al-Saif
Wilmington, Delaware, United States
Trial Status Not yet recruiting
Mahim Jain
Chicago, Illinois, United States
Trial Status Recruiting
Carlos Prada
Baltimore, Maryland, United States
Trial Status Recruiting
Julie Hoover-Fong
Cincinnati, Ohio, United States
Trial Status Recruiting
Benjamin Cocanougher
Philadelphia, Pennsylvania, United States
Trial Status Not yet recruiting
David Weber
Dallas, Texas, United States
Trial Status Not yet recruiting
Nadia Merchant
Houston, Texas, United States
Trial Status Recruiting
Carlos Bacino
San Antonio, Texas, United States
Trial Status Recruiting
Daniel Katselnik
Parkville, Victoria, Australia
Trial Status Recruiting
Ravi Savarirayan
Montreal, Quebec, Canada
Trial Status Not yet recruiting
Philippe Campeau
Genova, Italy
Trial Status Not yet recruiting
Mohamed Maghnie
Hokkaido, Japan
Trial Status Not yet recruiting
Shuntaro Morikawa
Osaka, Japan
Trial Status Recruiting
Jun Mori
Tokyo, Japan
Trial Status Recruiting
Kenichi Kashimada
Wroclaw, Poland
Trial Status Not yet recruiting
Robert Shanislaw Smigiel
Bucharest, Romania
Trial Status Not yet recruiting
Iuliana Gherlan
Craiova, Romania
Trial Status Not yet recruiting
Ioana Streata
Seoul, South Korea
Trial Status Recruiting
Jung Min Ko
Yangsan, South Korea
Trial Status Not yet recruiting
Chong-Kun Cheon
Bristol, United Kingdom
Trial Status Recruiting
Toby Candler
Glasgow, United Kingdom
Trial Status Recruiting
Helen McDevitt
Liverpool, United Kingdom
Trial Status Recruiting
Sharanya Ramakrishnan
London, United Kingdom
Trial Status Not yet recruiting
Moira Cheung
Manchester, United Kingdom
Trial Status Recruiting
Mars Skae
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
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
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