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

Last updated: January 17, 2025
Sponsor: BioMarin Pharmaceutical
Overall Status: Active - Recruiting

Phase

2

Condition

Idiopathic Short Stature

Severe Short Stature

Treatment

Placebo

Vosoritide Injection

Human Growth Hormone

Clinical Study ID

NCT06382155
111-210
  • Ages 3-10
  • All Genders

Study Summary

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), in children with idiopathic short stature (ISS).

Eligibility Criteria

Inclusion

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. Tanner Stage 1, at time of signing the ICF (unless too young to stage).

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)

  2. Previous treatment with a growth promoting agent

Study Design

Total Participants: 100
Treatment Group(s): 3
Primary Treatment: Placebo
Phase: 2
Study Start date:
October 21, 2024
Estimated Completion Date:
December 31, 2036

Study Description

Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.

Connect with a study center

  • Center Of Excellence in Diabetes and Endocrinology

    Sacramento, California 95821
    United States

    Active - Recruiting

  • Lundquist Institute for Biomedical Innovation (LA BioMed)

    Torrance, California 90502
    United States

    Active - Recruiting

  • Nemours Children's Health System - Corporate Headquarters

    Pensacola, Florida 32514
    United States

    Active - Recruiting

  • Centricity Research

    Columbus, Georgia 31904
    United States

    Active - Recruiting

  • St. Luke's Children's Endocrinology

    Boise, Idaho 83712
    United States

    Active - Recruiting

  • Rocky Mountain Clinical Research - Idaho Falls

    Idaho Falls, Idaho 83404
    United States

    Active - Recruiting

  • Children's Hospital at Montefiore

    Bronx, New York 10467
    United States

    Active - Recruiting

  • UBMD Pediatrics

    Buffalo, New York 14203
    United States

    Active - Recruiting

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