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

NCT06382155 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 111-210
• Study Type: interventional
• Target: 100
• Locations: 6 countries
• Sites: 47

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

Outcome Measures

Primary Outcomes (3)

• Change from baseline in Annualized Growth Velocity (AGV)

  At 6 months

• Change from baseline in height

  At 4 years

• Change from baseline in height Z-score

  At 4 years

Secondary Outcomes (18)

• Incidence of treatment-emergent adverse events

  Until the end of the study, up to 15 years

• Change from baseline in Height Z-score (average stature reference)

  At 6 months

• Change from baseline in height

  Every 6 months through the end of study, up to 15 years

• Change from baseline in height Z score

  Every 6 months through the end of study, up to 15 years

• Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)

  Every 6 months through the end of study, up to 15 years

Study Arms (5)

Vosoritide Dose 1 - Low Dose

EXPERIMENTAL

Vosoritide Dose 1 daily injection

Drug: Vosoritide Injection

Vosoritide Dose 2 - Medium Dose

EXPERIMENTAL

Vosoritide Dose 2 daily injection

Drug: Vosoritide Injection

Vosoritide Dose 3 - High Dose

EXPERIMENTAL

Vosoritide Dose 3 daily injection

Drug: Vosoritide Injection

Placebo

PLACEBO COMPARATOR

Placebo daily injection

Drug: Placebo

Human Growth Hormone

ACTIVE COMPARATOR

hGH daily injection (US only)

Drug: Human Growth Hormone

Interventions

Vosoritide Injection DRUG

Experimental Drug Lyophilized powder for reconstitution

Vosoritide Dose 1 - Low Dose; Vosoritide Dose 2 - Medium Dose; Vosoritide Dose 3 - High Dose

Human Growth Hormone DRUG

Commercial product containing somatotropin

Human Growth Hormone

Placebo DRUG

Lyophilized powder for reconstitution

Placebo

Eligibility Criteria

• Age: 3 Years - 11 Years
• Gender Eligibility Details: Participants must be ≥ 3 years old, and \< 10 years old (female) or \< 11 years old (males) at the time of signing the informed consent form (ICF).
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• Tanner Stage 1, at time of signing the ICF (unless too young to stage).
• 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)
• Previous treatment with a growth promoting agent

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (47)

Consano Clinical Research, LLC

California City, California, 78231, United States

RECRUITING

Rady Children's Hospital - San Diego

California City, California, 92123, United States

RECRUITING

Children's Hospital of Orange County Main Campus - Orange

California City, California, 92868, United States

RECRUITING

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Children's Hospital Oakland Research Institute

Oakland, California, 94609, United States

RECRUITING

Center Of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821, United States

RECRUITING

Lundquist Institute for Biomedical Innovation (LA BioMed)

Torrance, California, 90502, United States

RECRUITING

The Children's National Research Institute

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

RECRUITING

Nemours Children's Specialty Care Jacksonville

Jacksonville, Florida, 32207, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Optimal Research Sites

Orange City, Florida, 32763, United States

RECRUITING

Nemours Children's Health System - Corporate Headquarters

Pensacola, Florida, 32514, United States

RECRUITING

Centricity Research

Columbus, Georgia, 31904, United States

RECRUITING

St. Luke's Children's Endocrinology

Boise, Idaho, 83712, United States

RECRUITING

Rocky Mountain Clinical Research - Idaho Falls

Idaho Falls, Idaho, 83404, United States

RECRUITING

University of Iowa Stead Family Children's Hospital

Iowa City, Iowa, 52242, United States

RECRUITING

Kentucky Children's Hospital

Lexington, Kentucky, 40536, United States

RECRUITING

University of Louisville School of Medicine

Louisville, Kentucky, 40202, United States

RECRUITING

Velocity Clinical Research - MedPharmics - Lafayette

Lafayette, Louisiana, 70508, United States

RECRUITING

Ochsner Health Center for Children - New Orleans

New Orleans, Louisiana, 70121, United States

WITHDRAWN

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, 55454, United States

RECRUITING

UBMD Pediatrics

Buffalo, New York, 14203, United States

RECRUITING

NYU Langone Ambulatory Care Garden City

Garden City, New York, 11530, United States

WITHDRAWN

Atlantic Health Children's Specialty Center at Morristown

New York, New York, 07960, United States

RECRUITING

The Clinical and Translational Science Institute

New York, New York, 10016, United States

RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

RECRUITING

University of North Carolina Children's Specialty Clinic

Chapel Hill, North Carolina, 27514, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Prisma Health Children's Hospital - Midlands

Columbia, South Carolina, 29203, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Cook Children's Endocrinology

Fort Worth, Texas, 76104, United States

RECRUITING

Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

RECRUITING

Centre Hospitalier Universitaire d'Angers

Angers, 49 933, France

RECRUITING

Hôpital de la Timone - Enfants

Marseille, 13005, France

RECRUITING

CHU de Toulouse - Hôpital des Enfants

Toulouse, 31059, France

RECRUITING

Hôpital Bicêtre

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

RECRUITING

Universitätsklinikum des Saarlandes

Homburg, Saarland, 66421, Germany

RECRUITING

Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer

Florence, Florence, 50139, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù

Roma, Rome, 00165, Italy

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

RECRUITING

Seoul National University Bundang Hospital

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

RECRUITING

Inha University Hospital

Incheon, 22332, South Korea

RECRUITING

Chungnam National University Sejong Hospital (CNUSH)

Sejong, 30099, South Korea

RECRUITING

MeSH Terms

Interventions

vosoritide; Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Medical Director MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Central Study Contacts

Trial Specialist

CONTACT

+1 415.475.5852; medinfo@bmrn.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 19, 2024
• First Posted: April 24, 2024
• Study Start: October 21, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): December 1, 2036
• Last Updated: March 13, 2026

Record last verified: 2026-03

Locations

US (35); AU (1); DE (1); IT (3); FR (4); KR (3)