Recruiting
Phase 2

Vosoritide

Sponsor:

BioMarin Pharmaceutical

Code:

NCT06382155

Conditions

Idiopathic Short Stature

Eligibility Criteria

Sex: All

Age: 3 - 11

Healthy Volunteers: Not accepted

Interventions

Vosoritide Injection

Human Growth Hormone

Placebo

Study Details

Brief 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).

Conditions

Idiopathic Short Stature

Study ID

NCT06382155

Start date

Oct 21, 2024

Status verified date

May, 2026

Completion date

Dec, 2036

Anticipated

Primary completion date

Jun, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 3 - 11

Healthy Volunteers: Not accepted

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 Design

Enrollment

100 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Vosoritide Dose 1 - Low Dose

Vosoritide Dose 1 daily injection

experimental: Vosoritide Dose 2 - Medium Dose

Vosoritide Dose 2 daily injection

experimental: Vosoritide Dose 3 - High Dose

Vosoritide Dose 3 daily injection

placebo comparator: Placebo

Placebo daily injection

active comparator: Human Growth Hormone

hGH daily injection (US only)

Interventions

Vosoritide Injection

Experimental Drug Lyophilized powder for reconstitution

Human Growth Hormone

Commercial product containing somatotropin

Placebo

Lyophilized powder for reconstitution

Primary outcome measure

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

Central Contacts and Locations

Central contacts

Trial Specialist

+1 415.475.5852medinfo@bmrn.com

Locations

Consano Clinical Research, LLC

Recruiting

California City, California, United States, 78231

Contacts

Daniel Katselnik

210-545-4900katselnik.MD@ConsanoSA.com

Rady Children's Hospital - San Diego

Recruiting

California City, California, United States, 92123

Contacts

Mary (Marybeth) Patterson

858-966-4032mpatterson2@rchsd.org

Children's Hospital of Orange County Main Campus - Orange

Recruiting

California City, California, United States, 92868

Contacts

Amrit Bhangoo

+17145098634abhangoo@choc.org

Children's Hospital Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

Anna Ryabets-Lienhard

323-361-6478aryabets@chla.usc.edu

Children's Hospital Oakland Research Institute

Recruiting

Oakland, California, United States, 94609

Contacts

Dr Hannah Chesser

hannah.chesser@ucsf.edu

Center Of Excellence in Diabetes and Endocrinology

Recruiting

Sacramento, California, United States, 95821

Contacts

Dr Gnanagurudasan Prakasam

916-570-2756Gnanagurudasan.Prakasam@sutterhealth.org

Lundquist Institute for Biomedical Innovation (LA BioMed)

Recruiting

Torrance, California, United States, 90502

Contacts

Dr Lynda Polgreen

3102221961lpolgreen@lundquist.org

The Children's National Research Institute

Recruiting

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

Contacts

Dr Andrew Dauber

adauber@childrensnational.org

Nemours Children's Specialty Care Jacksonville

Recruiting

Jacksonville, Florida, United States, 32207

Contacts

Dr Larry Fox

larry.fox@nemours.org

Nicklaus Children's Hospital

Recruiting

Miami, Florida, United States, 33155

Contacts

Dr Adriana Carrillo Iregui

adriana.carrillo@nicklaushealth.org

Optimal Research Sites

Recruiting

Orange City, Florida, United States, 32763

Contacts

Dr Jaqueline Freyre-Cubano

386-218-5911jfreyre@optimalresearchsites.net

Nemours Children's Health System - Corporate Headquarters

Recruiting

Pensacola, Florida, United States, 32514

Contacts

Dr Jean-Claude Desmangles

850-505-4793jean-claude.desmangles@nemours.ort

Centricity Research

Recruiting

Columbus, Georgia, United States, 31904

Contacts

Steven Leichter Principal Investigator

833-3235646sleichter@centricityresearch.com

Rocky Mountain Clinical Research - Idaho Falls

Recruiting

Idaho Falls, Idaho, United States, 83404

Contacts

Dr Joshua Smith

208-522-6005jms-research@idahomed.com

University of Iowa Stead Family Children's Hospital

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Dr Michael Tansey

(319) 356-4443michael-tansey@uiowa.edu

Kentucky Children's Hospital

Recruiting

Lexington, Kentucky, United States, 40536

Contacts

Dr Yuri Zarate

859-257-1000yuri.zarate@uky.edu

University of Louisville School of Medicine

Recruiting

Louisville, Kentucky, United States, 40202

Contacts

Dr Catalina Cabrera Salcedo

catalina.salcedo@louisville.edu

Velocity Clinical Research - MedPharmics - Lafayette

Recruiting

Lafayette, Louisiana, United States, 70508

Contacts

Dr Jibran Atwi

(337) 519-4742jiatwi@velocityclinical.com

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Dr Inas H. Thomas

734-674-5175inash@med.umich.edu

University of Minnesota Masonic Children's Hospital

Recruiting

Minneapolis, Minnesota, United States, 55454

Contacts

Dr Bradley Miller

612-626-2916mille685@umn.edu

UBMD Pediatrics

Recruiting

Buffalo, New York, United States, 14203

Contacts

Dr Teresa Quattrin

716-323-0170tquattrin@upa.chob.edu

Atlantic Health Children's Specialty Center at Morristown

Recruiting

New York, New York, United States, 07960

Contacts

Dr Lawrence Silverman

973-971-4340lawrence.silverman@atlantichealth.org

The Clinical and Translational Science Institute

Recruiting

New York, New York, United States, 10016

Contacts

Dr Emily Breidbart

(212) 263-5290emily.breidbart@nyulangone.org

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Robert Rapaport

(212) 241-8523robert.rapaport@mountsinai.org

Children's Hospital at Montefiore

Recruiting

The Bronx, New York, United States, 10467

Contacts

Dr Laurie Cohen

(718)920-4664lacohen@montefiore.org

University of North Carolina Children's Specialty Clinic

Recruiting

Chapel Hill, North Carolina, United States, 27514

Contacts

Nina Jain

919-718-9512nina_jain@med.unc.edu

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Dr Gajanthan Muthuvel

(513) 636-8444gajanthan.muthuvel@cchmc.org

Prisma Health Children's Hospital - Midlands

Recruiting

Columbia, South Carolina, United States, 29203

Contacts

Dr Alison Lunsford

803-434-7990alison.lunsford@prismahealth.org

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75390

Contacts

Dr Nadia Merchant

214-456-5959nadia.merchant@utsouthwestern.edu

Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77030

Contacts

Dr Lin Yuezhen

yuezhenl@bcm.edu

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Dr Patricia Fechner

206-884-8540patricia.fechner@seattlechildrens.org

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

May 22, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by BioMarin Pharmaceutical on 2026-05-22.