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.
Ready to participate?

Review the details below, then apply to join this clinical 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
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
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
Loading…

Loading trial locations…

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

Trial Specialist

1-800-983-4587

medinfo@bmrn.com

CONTACT