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

Studying vosoritide for kids with unexplained short stature.

Recruiting
3-10 years
All
Phase 2

This study looks at the effects of a medicine called *vosoritide* on children with **idiopathic short stature (ISS)**. ISS means being shorter than other kids of the same age for no known reason. The study compares vosoritide with human growth hormone (hGH), which is another treatment for short stature. First, there is a **6-month observation period** to see how much the children grow naturally. After that, children may receive vosoritide or a fake treatment (placebo) for another 6 months. Some children will also receive hGH treatment for at least 4 years. **Vosoritide** is given until the child reaches their almost final adult height or until they are 16 years old for girls and 18 years old for boys, whichever comes later.

  • Study lasts several years, depending on growth.
  • Involves a minimum of 6 months on vosoritide or placebo.
  • Requires frequent visits for growth checks and treatment.

Children must be at a certain height level and cannot have other genetic conditions affecting height to participate. It's important to discuss potential benefits and risks with the doctor before joining.

Study details
    Idiopathic Short Stature

NCT06382155

BioMarin Pharmaceutical

25 January 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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