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

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Trial Status Not Yet Recruiting

Trial Identifier

NCT07441876

Condition

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.

Trial 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 Criteria

Birth Sex

All

Age

2 Years to 17 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 333, Vosoritide Injection [Voxzogo]

Phase:

Phase 2/Phase 3

Study Type:

Interventional

Number of Participants:

160

Study Started:

2026-02

Study Updated:

2026-03-02

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

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