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Phase 2 randomized trial of vosoritide in infants and young children with hypochondroplasia
jRCT2061250054Phase 2Active, not recruiting
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Nov 2025
- Modality
- Recombinant Proteins and Enzymes, Peptides
01
Overview
Phase 2, randomised, double-blind trial in hypochondroplasia (HCH) comparing Vosoritide with Placebo (2 arms). Primary endpoints: Incidence of treatment-emergent adverse events (TEAEs), Incidence of serious adverse events (SAEs) and 3 more. Started Nov 2025; status: active, not recruiting.
02
Drugs
vosoritide
03
Conditions
hypochondroplasia (HCH)
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