Evaluating Vosoritide for Children with Short Stature

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the effects of vosoritide, a novel treatment, on children with idiopathic short stature (ISS) compared to human growth hormone (hGH). After an initial observational period to establish baseline growth, participants will be randomized to receive either vosoritide or placebo for at least six months, followed by open-label treatment until they reach near-final adult height. The study will also include a group receiving hGH for a minimum of four years to compare outcomes. The goal is to determine the efficacy and safety of vosoritide in promoting growth in affected children.

Why it matters

Who can join: the full rules

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

Where it's running

• Consano Clinical Research, LLC
  California City, California, United States Recruiting
• Rady Children's Hospital - San Diego
  California City, California, United States Recruiting
• Children's Hospital of Orange County Main Campus - Orange
  California City, California, United States Recruiting
• Children's Hospital Los Angeles
  Los Angeles, California, United States Recruiting
• Children's Hospital Oakland Research Institute
  Oakland, California, United States Recruiting
• Center Of Excellence in Diabetes and Endocrinology
  Sacramento, California, United States Recruiting

Study contacts

• Study coordinator: Trial Specialist
• Email: medinfo@bmrn.com
• Phone: +1 415.475.5852

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Evaluating the long-term safety and effectiveness of Growtropin®-II in children with short stature

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.