RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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), in children with idiopathic short stature (ISS).

Official Title

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

Quick Facts

Study Start:2024-10-21

Study Completion:2036-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06382155

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.

Ages Eligible for Study:3 Years to 10 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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). 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	Pregnancy or breastfeeding Severe psychiatric disorders Active substance abuse Unstable medical conditions Inability to comply with study requirements

Inclusion Criteria

Exclusion 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).
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

Pregnancy or breastfeeding
Severe psychiatric disorders
Active substance abuse
Unstable medical conditions
Inability to comply with study requirements

Contacts and Locations

Study Contact

Trial Specialist

CONTACT

+1 415.475.5852

medinfo@bmrn.com

Principal Investigator

Medical Director MD

STUDY_DIRECTOR

BioMarin Pharmaceutical

Study Locations (Sites)

Centricity Research

Columbus, Georgia, 31904

United States

St. Luke's Children's Endocrinology

Boise, Idaho, 83712

United States

Collaborators and Investigators

Sponsor: BioMarin Pharmaceutical

Medical Director MD, STUDY_DIRECTOR, 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 Registration Dates

Study Start Date2024-10-21

Study Completion Date2036-12

Study Record Updates

Study Start Date2024-10-21

Study Completion Date2036-12

Terms related to this study

Additional Relevant MeSH Terms

Idiopathic Short Stature