Actively Recruiting
Phase 2 Study of Vosoritide Treatment for Growth in Children With Noonan Syndrome Who Have Inadequate Growth After or During Human Growth Hormone Therapy
Led by BioMarin Pharmaceutical · Updated on 2026-05-11
30
Participants Needed
36
Research Sites
756 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying children with Noonan syndrome who have inadequate growth during or after treatment with human growth hormone hGH. The study aims to evaluate the effects of three different doses of vosoritide on growth, specifically looking at annualized growth velocity AGV after six months of treatment. The long-term safety and effectiveness of vosoritide at the therapeutic dose will be assessed up to when participants reach their final adult height FAH. This Phase 2, randomized, multicenter trial compares three dosing regimens of vosoritide, which is given as an injection. Participants receive one of three different doses of this experimental drug, provided as a lyophilized powder for reconstitution. The study includes both a short-term evaluation of growth changes over six months and a longer-term assessment of height and safety measures up to 15 years. During the study, children will have regular height measurements and assessments of growth velocity, body proportions, bone density, and bone age. Researchers will also monitor heart conditions, bone-related events, and quality of life using various questionnaires and physical activity scores. Blood and urine tests will track vosoritide levels and biological markers. Safety and growth will be followed every six to twelve months until participants reach their final adult height, which may take up to 15 years.
CONDITIONS
Brief Title
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 3 years old or older, and under 11 years old for females or under 12 years old for males, at the time of consent.
- Genetically confirmed diagnosis of Turner syndrome, SHOX deficiency, or Noonan syndrome.
- Height corresponding to a Z-score of -1.28 or lower (below the 10th percentile) for age and sex.
- Tanner Stage 1 at the time of consent.
- Previous or current treatment with human growth hormone for short stature related to their condition.
- Inadequate growth confirmed by an annualized growth velocity below the average for their age and sex.
You will not qualify if you...
- Turner syndrome participants with Y-chromosome material unless they have had gonadectomy and have fully external female genitalia.
- Diagnosis of other systemic diseases that may cause short stature besides Turner syndrome, SHOX deficiency, or Noonan syndrome.
- Bone age advanced more than 2 years beyond chronological age.
- Uncorrected congenital heart disease that increases risk during low blood pressure.
- Unstable condition likely to need surgical intervention during the study.
- Evidence of decreased growth velocity less than 1.5 cm/year over at least 6 months and growth plate closure.
- Previous, planned, or expected limb-lengthening surgery during the study.
- Planned or expected bone-related surgery during the study, excluding tooth extraction.
Research Team
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Trial Specialist
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Study Manager
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