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A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Researching Vosoritide for growth issues in kids with Noonan Syndrome.

Recruiting
3-11 years
All
Phase 2

This study is for kids with Noonan syndrome, a condition that affects growth. The aim is to see how well the medicine vosoritide helps kids grow after 6 months, especially if they didn't grow enough with human growth hormone (hGH) treatment. Vosoritide might help increase their adult height.

To join, kids need to be between 3 to 10 years old for girls and 3 to 11 years old for boys, and have had growth problems even after using hGH. They also need to have a genetic test confirming Noonan syndrome. Kids with other health issues or past surgeries that affect growth can't join.

  • **Study Length:** 6 months minimum, with possible longer follow-up.
  • **Visits:** Multiple visits to the clinic for assessments.
  • **Risks:** Possible side effects and need for monitoring, especially for heart health.

If your child fits the requirements and you are interested in the study, it might help them grow better with careful medical support.

Study details
    Noonan Syndrome

NCT06668805

BioMarin Pharmaceutical

23 May 2026

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