A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
ConditionHypochondroplasia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age0-36
SponsorBioMarin Pharmaceutical
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.
Eligibility criteria
Qualifiers
Participants must be 0 to < 36 months of age at randomization.
Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
Participants aged 0 to < 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to < 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
Disqualifiers
Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
Have an unstable medical condition likely to require surgical intervention during the study period.
Taking any of the prohibited medications.
Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time.
Trial design
Treatments tested in this trial
- Vosoritide
- Placebo
Treatment groups
60 Participants
are divided into 2 treatment groupsSponsors and collaborators
BioMarin Pharmaceutical
Lead sponsor
ICON Clinical Research
Collaborator
Source ClinicalTrials.gov