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Phase 3 Long-Term Extension Study of Vosoritide in Children With Hypochondroplasia
jRCT2051250157Phase 3Active, not recruiting
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Dec 2025
- Modality
- Recombinant Proteins and Enzymes, Peptides
01
Overview
Phase 3, open-label, single-arm trial of vosoritide in hypochondroplasia (HCH), sponsored by BioMarin Pharmaceutical Inc. Primary endpoints: Incidence of treatment emergent adverse events over time until final adult height, Change from baseline in height Z-score yearly until final adult height and 1 more. Started Dec 2025; status: active, not recruiting.
02
Drugs
vosoritide
03
Conditions
hypochondroplasia (HCH)
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