AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

← Trials
🧪

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

RecruitingPhase 2Ages 3 Years - 11 YearsAllBioMarin Pharmaceutical
💬

In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Key Inclusion Criteria: 1. 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 2. 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). Key Exclusions: 1. 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. 2. Previous treatment with a growth promoting agent
💊

What it tests

  • Vosoritide Injection
  • Human Growth Hormone
  • Placebo
📍

Where

47 sites
Consano Clinical Research, LLC
California City, United States
Rady Children's Hospital - San Diego
California City, United States
Children's Hospital of Orange County Main Campus - Orange
California City, United States
Children's Hospital Los Angeles
Los Angeles, United States
Children's Hospital Oakland Research Institute
Oakland, United States
Center Of Excellence in Diabetes and Endocrinology
Sacramento, United States
Lundquist Institute for Biomedical Innovation (LA BioMed)
Torrance, United States
The Children's National Research Institute
Washington D.C., United States

…and 39 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
🛡️

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy