Back to Results

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

Interested in this Trial?

Contact Us

Trial Status Recruiting

Trial Identifier

NCT06382155

Condition

Idiopathic Short Stature

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:

2025-01-20

Trial Locations

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

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).

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
Consent*
Consent*
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form

What’s Next?

Visit the following links to learn more about clinical trials.

Discuss this trial with your care team.

You may wish to print this page to share the details with your healthcare providers and discuss whether you may be eligible.

Print

Get in touch.

Contact Us