Health TrialsbyHealthPanel

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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • Vosoritide

    Drug

    The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH

  • Placebo

    Drug

    Subcutaneous injection of recommended dose of placebo

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: Vosoritide injection with vial and syringeExperimental treatment 1 intervention
Group B: Placebo injection with vial and syringePlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-emergent adverse events

Time frame
From baseline to end of treatment at 52 weeks
2

Incidence of serious adverse events versus placebo over the course of the study

Time frame
From baseline to end of treatment at 52 weeks
3

Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)

Time frame
At week 26, at week 52
4

Changes from baseline in heart rate

Units of measure: bpm

Time frame
At week 13, at week 26, at week 39, at week 52

Secondary outcomes

1

Change in height

Time frame
At week 52
2

Cumulative annualized growth velocity (AGV)

Time frame
At week 52
3

6-month interval AGV

Time frame
At week 26, at week 52
4

Change from baseline in upper to lower body segment ratio

Time frame
At week 52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

BioMarin Pharmaceutical

Lead sponsor

ICON Clinical Research

Collaborator

Source ClinicalTrials.gov
Apply now