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

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Trial Status Recruiting

Trial Identifier

NCT06382155

Condition

Idiopathic Short Stature

Study Resources

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

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

Eligibility Criteria

Birth Sex

All

Age

3 Years to 10 Years

Healthy Volunteers

No

Drug/Treatment:

Vosoritide Injection, Human Growth Hormone, Placebo

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

100

Study Started:

2024-10-21

Study Updated:

2025-03-25

Trial Locations

  • Center Of Excellence in Diabetes and Endocrinology

    Sacramento, California, United States

  • Lundquist Institute for Biomedical Innovation (LA BioMed)

    Torrance, California, United States

  • Nemours Children's Health System - Corporate Headquarters

    Pensacola, Florida, United States

  • Centricity Research

    Columbus, Georgia, United States

  • St. Luke's Children's Endocrinology

    Boise, Idaho, United States

  • Rocky Mountain Clinical Research - Idaho Falls

    Idaho Falls, Idaho, United States

  • Children's Hospital at Montefiore

    Bronx, New York, United States

  • UBMD Pediatrics

    Buffalo, New York, United States

Eligibility Criteria

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. Tanner Stage 1, at time of signing the ICF (unless too young to stage).

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)
2. Previous treatment with a growth promoting agent

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