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

May 20, 2026 updated by: BioMarin Pharmaceutical

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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.

A Treatment Completion visit will occur approximately 4 weeks after the last administration of investigational product. Participants who discontinue study treatment after ≥13 weeks of exposure may remain in the study and complete follow-up assessments per the protocol schedule (including annual and periodic assessments through End of Study). If a participant discontinues treatment and declines further participation, they will be asked to return for a final Treatment Completion and End of Study visit approximately 4 weeks after the last injection.

Safety monitoring includes regular clinical and imaging assessments, including hip and lower-extremity evaluations, monitoring for hypotension, fractures, and slipped capital femoral epiphysis, with oversight by an independent Data Monitoring Committee

An independent Data Monitoring Committee will periodically review safety data and may recommend treatment discontinuation based on predefined safety signals

Study Type

Interventional

Enrollment (Estimated)

100

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3052
  • France
      • Angers, France, 49 933
        • Recruiting
        • Centre Hospitalier Universitaire d'Angers
        • Contact:
      • Marseille, France, 13005
        • Recruiting
        • Hôpital de la Timone - Enfants
        • Contact:
      • Toulouse, France, 31059
        • Recruiting
        • CHU de Toulouse - Hôpital des Enfants
        • Contact:
    • Île-de-France Region
      • Le Kremlin-Bicêtre, Île-de-France Region, France, 94270
        • Recruiting
        • Hôpital Bicêtre
        • Contact:
  • Germany
    • Saarland
      • Homburg, Saarland, Germany, 66421
        • Recruiting
        • Universitatsklinikum des Saarlandes
        • Contact:
  • Italy
      • Genova, Italy, 16147
        • Recruiting
        • IRCCS Istituto Giannina Gaslini
        • Contact:
    • Florence
      • Florence, Florence, Italy, 50139
        • Recruiting
        • Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
        • Contact:
    • Rome
      • Roma, Rome, Italy, 00165
        • Recruiting
        • Ospedale Pediatrico Bambino Gesu
        • Contact:
  • South Korea
      • Incheon, South Korea, 22332
        • Recruiting
        • Inha University Hospital
        • Contact:
      • Sejong, South Korea, 30099
        • Recruiting
        • Chungnam National University Sejong Hospital (CNUSH)
        • Contact:
    • Gyeonggi-do
      • Seongnam-si, Gyeonggi-do, South Korea, 13620
        • Recruiting
        • Seoul National University Bundang Hospital
        • Contact:
  • United States
    • California
      • California City, California, United States, 78231
        • Recruiting
        • Consano Clinical Research, LLC
        • Contact:
      • California City, California, United States, 92123
        • Recruiting
        • Rady Children's Hospital - San Diego
        • Contact:
      • California City, California, United States, 92868
        • Recruiting
        • Children's Hospital of Orange County Main Campus - Orange
        • Contact:
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
      • Oakland, California, United States, 94609
        • Recruiting
        • Children's Hospital Oakland Research Institute
        • Contact:
      • Sacramento, California, United States, 95821
      • Torrance, California, United States, 90502
        • Recruiting
        • Lundquist Institute for Biomedical Innovation (LA BioMed)
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20012
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Recruiting
        • Nemours Children's Specialty Care Jacksonville
        • Contact:
      • Miami, Florida, United States, 33155
      • Orange City, Florida, United States, 32763
      • Pensacola, Florida, United States, 32514
        • Recruiting
        • Nemours Children's Health System - Corporate Headquarters
        • Contact:
    • Georgia
      • Columbus, Georgia, United States, 31904
    • Idaho
      • Boise, Idaho, United States, 83712
        • Withdrawn
        • St. Luke's Children's Endocrinology
      • Idaho Falls, Idaho, United States, 83404
        • Recruiting
        • Rocky Mountain Clinical Research - Idaho Falls
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa Stead Family Children's Hospital
        • Contact:
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • Kentucky Children's Hospital
        • Contact:
      • Louisville, Kentucky, United States, 40202
    • Louisiana
      • Lafayette, Louisiana, United States, 70508
        • Recruiting
        • Velocity Clinical Research - MedPharmics - Lafayette
        • Contact:
      • New Orleans, Louisiana, United States, 70121
        • Withdrawn
        • Ochsner Health Center for Children - New Orleans
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Recruiting
        • University of Minnesota Masonic Children's Hospital
        • Contact:
    • New York
      • Buffalo, New York, United States, 14203
        • Recruiting
        • UBMD Pediatrics
        • Contact:
      • Garden City, New York, United States, 11530
        • Withdrawn
        • NYU Langone Ambulatory Care Garden City
      • New York, New York, United States, 10029
      • New York, New York, United States, 07960
      • New York, New York, United States, 10016
        • Recruiting
        • The Clinical and Translational Science Institute
        • Contact:
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Children's Hospital at Montefiore
        • Contact:
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Recruiting
        • University of North Carolina Children's Specialty Clinic
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
    • South Carolina
      • Columbia, South Carolina, United States, 29203
    • Texas
      • Dallas, Texas, United States, 75390
      • Fort Worth, Texas, United States, 76104
        • Withdrawn
        • Cook Children's Endocrinology
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine
        • Contact:
    • Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Vosoritide Dose 1 - Low Dose
Vosoritide Dose 1 daily injection
Drug: Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Experimental: Vosoritide Dose 2 - Medium Dose
Vosoritide Dose 2 daily injection
Drug: Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Experimental: Vosoritide Dose 3 - High Dose
Vosoritide Dose 3 daily injection
Drug: Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Placebo Comparator: Placebo
Placebo daily injection
Drug: Placebo
Lyophilized powder for reconstitution
Active Comparator: Human Growth Hormone
hGH daily injection (US only)
Drug: Human Growth Hormone
Commercial product containing somatotropin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in Annualized Growth Velocity (AGV)
Time Frame: At 6 months
At 6 months
Change from baseline in height
Time Frame: At 4 years
At 4 years
Change from baseline in height Z-score
Time Frame: At 4 years
At 4 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-emergent adverse events
Time Frame: Until the end of the study, up to 15 years
Until the end of the study, up to 15 years
Change from baseline in height
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline in height Z score
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline at pre-specified timepoints in serum collagen X marker (CXM)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline in bone age minus chronological age at pre-specified timepoints
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMD Z-score
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline in total body (less head) bone mineral content (BMC)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMC
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Maximum concentration (Cmax) of vosoritide in plasma
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Elimination half-life of vosoritide (t½)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Apparent clearance of vosoritide
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Time vosoritide is present at maximum concentration (Tmax)
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years
Change from baseline in Height Z-score (average stature reference)
Time Frame: At 6 months
At 6 months
Change from baseline in whole body (less head) bone mineral density (BMD) Z-score
Time Frame: Every 6 months through the end of study, up to 15 years
Every 6 months through the end of study, up to 15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Investigators

  • Study Director: Medical Director MD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 21, 2024

Primary Completion (Estimated)

June 1, 2026

Study Completion (Estimated)

December 1, 2036

Study Registration Dates

First Submitted

April 19, 2024

First Submitted That Met QC Criteria

April 19, 2024

First Posted (Actual)

April 24, 2024

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 20, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 111-210

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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