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New hope for short kids: experimental drug takes on growth hormone

NCT ID NCT06382155

Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called vosoritide in 100 children with idiopathic short stature (short height with no known cause). The goal is to see if vosoritide can safely improve growth compared to standard growth hormone. Participants will receive either vosoritide or growth hormone, and their height and growth rate will be tracked over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 100 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2024

Expected to finish

Dec 2036

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 11 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

isolated short stature

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Idiopathic short stature

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    43 sites in 6 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Atlantic Health Children's Specialty Center at Morristown

    RECRUITING

    New York, New York, 07960, United States

  • Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

    RECRUITING

    Florence, Florence, 50139, Italy

  • Baylor College of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • CHU de Toulouse - Hôpital des Enfants

    RECRUITING

    Toulouse, 31059, France

  • Center Of Excellence in Diabetes and Endocrinology

    RECRUITING

    Sacramento, California, 95821, United States

  • Centre Hospitalier Universitaire d'Angers

    RECRUITING

    Angers, 49 933, France

  • Centricity Research

    RECRUITING

    Columbus, Georgia, 31904, United States

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Children's Hospital Oakland Research Institute

    RECRUITING

    Oakland, California, 94609, United States

  • Children's Hospital at Montefiore

    RECRUITING

    The Bronx, New York, 10467, United States

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

    RECRUITING

    California City, California, 92868, United States

  • Chungnam National University Sejong Hospital (CNUSH)

    RECRUITING

    Sejong, 30099, South Korea

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Consano Clinical Research, LLC

    RECRUITING

    California City, California, 78231, United States

  • Cook Children's Endocrinology

    WITHDRAWN

    Fort Worth, Texas, 76104, United States

  • Hôpital Bicêtre

    RECRUITING

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

  • Hôpital de la Timone - Enfants

    RECRUITING

    Marseille, 13005, France

  • IRCCS Istituto Giannina Gaslini

    RECRUITING

    Genova, 16147, Italy

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

  • Inha University Hospital

    RECRUITING

    Incheon, 22332, South Korea

  • Kentucky Children's Hospital

    RECRUITING

    Lexington, Kentucky, 40536, United States

  • Lundquist Institute for Biomedical Innovation (LA BioMed)

    RECRUITING

    Torrance, California, 90502, United States

  • Murdoch Children's Research Institute

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • NYU Langone Ambulatory Care Garden City

    WITHDRAWN

    Garden City, New York, 11530, United States

  • Nemours Children's Health System - Corporate Headquarters

    RECRUITING

    Pensacola, Florida, 32514, United States

  • Nemours Children's Specialty Care Jacksonville

    RECRUITING

    Jacksonville, Florida, 32207, United States

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

  • Ochsner Health Center for Children - New Orleans

    WITHDRAWN

    New Orleans, Louisiana, 70121, United States

  • Optimal Research Sites

    RECRUITING

    Orange City, Florida, 32763, United States

  • Ospedale Pediatrico Bambino Gesù

    RECRUITING

    Roma, Rome, 00165, Italy

  • Prisma Health Children's Hospital - Midlands

    RECRUITING

    Columbia, South Carolina, 29203, United States

  • Rady Children's Hospital - San Diego

    RECRUITING

    California City, California, 92123, United States

  • Rocky Mountain Clinical Research - Idaho Falls

    RECRUITING

    Idaho Falls, Idaho, 83404, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • Seoul National University Bundang Hospital

    RECRUITING

    Seongnam-si, Gyeonggi-do, 13620, South Korea

  • St. Luke's Children's Endocrinology

    WITHDRAWN

    Boise, Idaho, 83712, United States

  • The Children's National Research Institute

    RECRUITING

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

  • The Clinical and Translational Science Institute

    RECRUITING

    New York, New York, 10016, United States

  • UBMD Pediatrics

    RECRUITING

    Buffalo, New York, 14203, United States

  • University of Iowa Stead Family Children's Hospital

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Louisville School of Medicine

    RECRUITING

    Louisville, Kentucky, 40202, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota Masonic Children's Hospital

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

  • University of North Carolina Children's Specialty Clinic

    RECRUITING

    Chapel Hill, North Carolina, 27514, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • Universitätsklinikum des Saarlandes

    RECRUITING

    Homburg, Saarland, 66421, Germany

  • Velocity Clinical Research - MedPharmics - Lafayette

    RECRUITING

    Lafayette, Louisiana, 70508, United States

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