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

RecruitingPhase 2/3

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Who can join

Ages 2 to 17 · All sexes

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

About the study

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.

What is being tested

Sponsor: BioMarin Pharmaceutical · Participants: 160 · Started: Apr 20, 2026

Contact the study team

Official record on ClinicalTrials.gov — NCT07441876

Locations in the U.S.

CaliforniaUCSF Benioff Children's Hospital Oakland, Oakland
DelawareNemours Children's Health, Wilmington (Not yet recruiting)
IllinoisAnn & Robert H. Lurie Children's Hospital of Chicago, Chicago
MarylandJohns Hopkins Medicine, Baltimore
OhioCincinnati Children's Hospital Medical Center, Cincinnati
PennsylvaniaChildren's Hospital of Philadelphia, Philadelphia
TexasTexas Children's Hospital, Dallas
Texas Children Hospital, Baylor College of Medicine, Houston
Consano Clinical Research, LLC, San Antonio

Conditions

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.