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Clinical Trials in the USA / NCT06382155
Recruiting Phase 2

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

NCT06382155 · tracked via the Priya Life Science USA tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2024-10-21
Last updated
2026-05-22

Condition(s) studied

Idiopathic Short Stature

Investigational drug(s) / intervention(s)

Vosoritide InjectionHuman Growth HormonePlacebo

Vosoritide Injection: Experimental Drug Lyophilized powder for reconstitution

Human Growth Hormone: Commercial product containing somatotropin

Placebo: Lyophilized powder for reconstitution

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)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Eligibility

Sex
ALL
Min age
3 Years
Max age
11 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (47)

FacilityCityRegionStatus
Consano Clinical Research, LLC California City California Recruiting
Rady Children's Hospital - San Diego California City California Recruiting
Children's Hospital of Orange County Main Campus - Orange California City California Recruiting
Children's Hospital Los Angeles Los Angeles California Recruiting
Children's Hospital Oakland Research Institute Oakland California Recruiting
Center Of Excellence in Diabetes and Endocrinology Sacramento California Recruiting
Lundquist Institute for Biomedical Innovation (LA BioMed) Torrance California Recruiting
The Children's National Research Institute Washington D.C. District of Columbia Recruiting
Nemours Children's Specialty Care Jacksonville Jacksonville Florida Recruiting
Nicklaus Children's Hospital Miami Florida Recruiting
Optimal Research Sites Orange City Florida Recruiting
Nemours Children's Health System - Corporate Headquarters Pensacola Florida Recruiting
Centricity Research Columbus Georgia Recruiting
St. Luke's Children's Endocrinology Boise Idaho Withdrawn
Rocky Mountain Clinical Research - Idaho Falls Idaho Falls Idaho Recruiting
University of Iowa Stead Family Children's Hospital Iowa City Iowa Recruiting
Kentucky Children's Hospital Lexington Kentucky Recruiting
University of Louisville School of Medicine Louisville Kentucky Recruiting
Velocity Clinical Research - MedPharmics - Lafayette Lafayette Louisiana Recruiting
Ochsner Health Center for Children - New Orleans New Orleans Louisiana Withdrawn
University of Michigan Ann Arbor Michigan Recruiting
University of Minnesota Masonic Children's Hospital Minneapolis Minnesota Recruiting
UBMD Pediatrics Buffalo New York Recruiting
NYU Langone Ambulatory Care Garden City Garden City New York Withdrawn
Atlantic Health Children's Specialty Center at Morristown New York New York Recruiting
The Clinical and Translational Science Institute New York New York Recruiting
Icahn School of Medicine at Mount Sinai New York New York Recruiting
Children's Hospital at Montefiore The Bronx New York Recruiting
University of North Carolina Children's Specialty Clinic Chapel Hill North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
Prisma Health Children's Hospital - Midlands Columbia South Carolina Recruiting
University of Texas Southwestern Medical Center Dallas Texas Recruiting
Cook Children's Endocrinology Fort Worth Texas Withdrawn
Baylor College of Medicine Houston Texas Recruiting
Seattle Children's Hospital Seattle Washington Recruiting
Murdoch Children's Research Institute Parkville Victoria Recruiting
Centre Hospitalier Universitaire d'Angers Angers France Recruiting
Hôpital de la Timone - Enfants Marseille France Recruiting
CHU de Toulouse - Hôpital des Enfants Toulouse France Recruiting
Hôpital Bicêtre Le Kremlin-Bicêtre Île-de-France Region Recruiting

+ 7 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06382155 on ClinicalTrials.gov ↗ ← All trials in the USA