A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3-11
SponsorBioMarin Pharmaceutical

About this trial

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Eligibility criteria

Qualifiers

Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts

If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs).

Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency.

Previous treatment with a growth promoting agent

Disqualifiers

None

Trial design

Treatments tested in this trial

  • Vosoritide Injection
  • Human Growth Hormone
  • Placebo

Treatment groups

100 Participants
are divided into 5 treatment groups

Locations

47
Australia
Murdoch Children's Research Institute3052, ParkvilleVictoria, Australia
France
Hôpital Bicêtre94270, Le Kremlin-BicêtreÎle-de-France Region, France
Centre Hospitalier Universitaire d'Angers49 933, Angers France
Hôpital de la Timone - Enfants13005, Marseille France

Sponsors and collaborators

BioMarin Pharmaceutical

Lead sponsor