RecruitingPhase 2/3

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

United States, Australia160 participantsStarted 2026-04

Plain-language 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.

Who can participate

Age range2 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

Exclusion criteria

✕. 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

What they're measuring

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

Timeframe: 52 weeks

Phase 3: Annualized Growth Velocity (AGV) at Week 52

Timeframe: 52 weeks

Trial details

NCT IDNCT07441876

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-06

Contact for this trial

Trial Specialist

1-800-983-4587 medinfo@bmrn.com

View on ClinicalTrials.gov More Achondroplasia trials