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

Recruiting Phase 2 Phase 3 Interventional Study
Achondroplasia
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
2 – 17
Sex
Any
Trial phase
Phase 2/3
Study type
Interventional
Purpose
Treatment
Participants needed
160 (estimated)
Sponsor
BioMarin Pharmaceutical · Industry
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About This Trial

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.

Trial Locations
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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 (…
Contacts

Trial Specialist

1-800-983-4587

medinfo@bmrn.com

CONTACT

Study Manager

1-800-983-4587

Medinfo@bmrn.com

CONTACT