Home › Trials › NCT06382155

Evaluating Vosoritide for Children with Short Stature

A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature

Phase 2 Interventional BioMarin Pharmaceutical · NCT06382155

This study is testing a new treatment called vosoritide to see if it helps children with short stature grow better than the standard growth hormone treatment.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment100 (estimated)
Ages3 Years to 11 Years
SexAll
SponsorBioMarin Pharmaceutical Industry-sponsored
Locations47 sites (California City, California and 46 other locations)
Trial IDNCT06382155 on ClinicalTrials.gov

What this trial studies

This study aims to assess the effects of vosoritide, a novel treatment, on children with idiopathic short stature (ISS) compared to human growth hormone (hGH). After an initial observational period to establish baseline growth, participants will be randomized to receive either vosoritide or placebo for at least six months, followed by open-label treatment until they reach near-final adult height. The study will also include a group receiving hGH for a minimum of four years to compare outcomes. The goal is to determine the efficacy and safety of vosoritide in promoting growth in affected children.

Who should consider this trial

Good fit: Ideal candidates are children with a height Z-score of ≤ -2.25 SDs and Tanner Stage 1 at the time of consent.

Not a fit: Patients with known genetic causes of short stature or previous treatment with growth-promoting agents may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve growth outcomes for children with idiopathic short stature.

How similar studies have performed: Previous studies have shown promise in using growth hormone therapies for similar conditions, but the approach with vosoritide is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

California City, California and 46 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Idiopathic Short Stature
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.