A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Recruiting
Phase 2
Interventional Study
Hypochondroplasia
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 0 Months – 36 Months
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 60 (estimated)
- Sponsor
- BioMarin Pharmaceutical · Industry
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About This Trial
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
1. Participants must be 0 to \< 36 months of age at randomization.
2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
3. Participants aged 0 to \< 12 months must h…
Contacts