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

RecruitingPhase 2

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

Who can join

Ages 3 to 11 · All sexes

Full eligibility 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

About the study

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).

What is being tested

Sponsor: BioMarin Pharmaceutical · Participants: 100 · Started: Oct 21, 2024

Contact the study team

Official record on ClinicalTrials.gov — NCT06382155

Locations in the U.S.

CaliforniaChildren's Hospital of Orange County Main Campus - Orange, California City
Consano Clinical Research, LLC, California City
Rady Children's Hospital - San Diego, California City
Children's Hospital Los Angeles, Los Angeles
Children's Hospital Oakland Research Institute, Oakland
Center Of Excellence in Diabetes and Endocrinology, Sacramento
Lundquist Institute for Biomedical Innovation (LA BioMed), Torrance
District of ColumbiaThe Children's National Research Institute, Washington D.C.
FloridaNemours Children's Specialty Care Jacksonville, Jacksonville
Nicklaus Children's Hospital, Miami
Optimal Research Sites, Orange City
Nemours Children's Health System - Corporate Headquarters, Pensacola
GeorgiaCentricity Research, Columbus
IdahoRocky Mountain Clinical Research - Idaho Falls, Idaho Falls
IowaUniversity of Iowa Stead Family Children's Hospital, Iowa City
KentuckyKentucky Children's Hospital, Lexington
University of Louisville School of Medicine, Louisville
LouisianaVelocity Clinical Research - MedPharmics - Lafayette, Lafayette
MichiganUniversity of Michigan, Ann Arbor
MinnesotaUniversity of Minnesota Masonic Children's Hospital, Minneapolis
New YorkUBMD Pediatrics, Buffalo
Atlantic Health Children's Specialty Center at Morristown, New York
Icahn School of Medicine at Mount Sinai, New York
The Clinical and Translational Science Institute, New York
Children's Hospital at Montefiore, The Bronx
North CarolinaUniversity of North Carolina Children's Specialty Clinic, Chapel Hill
OhioCincinnati Children's Hospital Medical Center, Cincinnati
South CarolinaPrisma Health Children's Hospital - Midlands, Columbia
TexasUniversity of Texas Southwestern Medical Center, Dallas
Baylor College of Medicine, Houston
WashingtonSeattle Children's Hospital, Seattle

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.