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

Last updated: March 12, 2026
Sponsor: BioMarin Pharmaceutical
Overall Status: Active - Recruiting

Phase

2

Condition

Severe Short Stature

Idiopathic Short Stature

Treatment

Placebo

Vosoritide Injection

Human Growth Hormone

Clinical Study ID

NCT06382155
111-210
  • Ages 3-11
  • 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)(hGH; only in the United States), 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

  • Murdoch Children's Research Institute

    Parkville, Victoria 3052
    Australia

    Active - Recruiting

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, Île-de-France Region 94270
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire d'Angers

    Angers, 49 933
    France

    Active - Recruiting

  • Hôpital de la Timone - Enfants

    Marseille, 13005
    France

    Active - Recruiting

  • CHU de Toulouse - Hôpital des Enfants

    Toulouse, 31059
    France

    Active - Recruiting

  • Universitätsklinikum des Saarlandes

    Homburg, Saarland 66421
    Germany

    Active - Recruiting

  • Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

    Florence, Florence 50139
    Italy

    Active - Recruiting

  • Ospedale Pediatrico Bambino Gesù

    Roma, Rome 00165
    Italy

    Active - Recruiting

  • IRCCS Istituto Giannina Gaslini

    Genova, 16147
    Italy

    Active - Recruiting

  • Seoul National University Bundang Hospital

    Seongnam-si, Gyeonggi-do 13620
    South Korea

    Active - Recruiting

  • Inha University Hospital

    Incheon, 22332
    South Korea

    Active - Recruiting

  • Chungnam National University Sejong Hospital (CNUSH)

    Sejong, 30099
    South Korea

    Active - Recruiting

  • Children's Hospital of Orange County Main Campus - Orange

    California City, California 92868
    United States

    Active - Recruiting

  • Consano Clinical Research, LLC

    California City, California 78231
    United States

    Active - Recruiting

  • Rady Children's Hospital - San Diego

    California City, California 92123
    United States

    Active - Recruiting

  • Children's Hospital Los Angeles

    Los Angeles, California 90027
    United States

    Active - Recruiting

  • Children's Hospital Oakland Research Institute

    Oakland, California 94609
    United States

    Active - Recruiting

  • 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

  • The Children's National Research Institute

    Washington D.C., District of Columbia 20012
    United States

    Active - Recruiting

  • Nemours Children's Specialty Care Jacksonville

    Jacksonville, Florida 32207
    United States

    Active - Recruiting

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Active - Recruiting

  • Optimal Research Sites

    Orange City, Florida 32763
    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

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa 52242
    United States

    Active - Recruiting

  • Kentucky Children's Hospital

    Lexington, Kentucky 40536
    United States

    Active - Recruiting

  • University of Louisville School of Medicine

    Louisville, Kentucky 40202
    United States

    Active - Recruiting

  • Velocity Clinical Research - MedPharmics - Lafayette

    Lafayette, Louisiana 70508
    United States

    Active - Recruiting

  • Ochsner Health Center for Children - New Orleans

    New Orleans, Louisiana 70121
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Active - Recruiting

  • University of Minnesota Masonic Children's Hospital

    Minneapolis, Minnesota 55454
    United States

    Active - Recruiting

  • Children's Hospital at Montefiore

    Bronx, New York 10467
    United States

    Site Not Available

  • UBMD Pediatrics

    Buffalo, New York 14203
    United States

    Active - Recruiting

  • NYU Langone Ambulatory Care Garden City

    Garden City, New York 11530
    United States

    Site Not Available

  • Atlantic Health Children's Specialty Center at Morristown

    New York, New York 07960
    United States

    Active - Recruiting

  • Icahn School of Medicine at Mount Sinai

    New York, New York 10029
    United States

    Active - Recruiting

  • The Clinical and Translational Science Institute

    New York, New York 10016
    United States

    Active - Recruiting

  • Children's Hospital at Montefiore

    The Bronx, New York 10467
    United States

    Active - Recruiting

  • University of North Carolina Children's Specialty Clinic

    Chapel Hill, North Carolina 27514
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • Prisma Health Children's Hospital - Midlands

    Columbia, South Carolina 29203
    United States

    Active - Recruiting

  • University of Texas Southwestern Medical Center

    Dallas, Texas 75390
    United States

    Active - Recruiting

  • Cook Children's Endocrinology

    Fort Worth, Texas 76104
    United States

    Active - Recruiting

  • Baylor College of Medicine

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Active - Recruiting

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