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Evaluating vosoritide for growth in children with specific genetic conditions

A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Phase 2 Interventional BioMarin Pharmaceutical · NCT06668805

This study is testing if a new growth medication called vosoritide can help boys and girls with certain genetic conditions grow better than those who are only using standard growth hormone treatment.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment30 (estimated)
Ages3 Years to 11 Years
SexAll
SponsorBioMarin Pharmaceutical Industry-sponsored
Locations36 sites (Los Angeles, California and 35 other locations)
Trial IDNCT06668805 on ClinicalTrials.gov

What this trial studies

This Phase 2 basket study aims to assess the efficacy and safety of vosoritide in children aged 3 to 10 (females) or 3 to 11 (males) with Turner syndrome, SHOX deficiency, or Noonan syndrome who have not responded adequately to human growth hormone (hGH). Participants will receive one of three doses of vosoritide or continue with hGH for a period of 6 months, with growth measured by annual growth velocity (AGV). The study will also evaluate the long-term effects of vosoritide on final adult height over a 2-year period. This multicenter approach allows for a diverse patient population to be included in the analysis.

Who should consider this trial

Good fit: Ideal candidates are children aged 3 to 10 (females) or 3 to 11 (males) with a confirmed diagnosis of Turner syndrome, SHOX deficiency, or Noonan syndrome and an inadequate response to prior hGH treatment.

Not a fit: Patients with systemic diseases or those who have Y-chromosome material in Turner syndrome without prior gonadectomy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve growth outcomes for children with these genetic conditions who do not respond well to existing therapies.

How similar studies have performed: While this approach is novel in the context of these specific conditions, similar studies evaluating vosoritide have shown promise in other contexts.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form
2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex.
4. Tanner Stage 1, at time of signing the ICF.
5. Previous or current hGH treatment for short stature associated with their condition.
6. Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts

Exclusion Criteria:

1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.
3. Bone age advanced beyond chronological age by more than 2 years.
4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
5. Have an unstable condition likely to require surgical intervention during the study.
6. Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays.
7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.
8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Where this trial is running

Los Angeles, California and 35 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Noonan SyndromeShort StatureMusculoskeletal DiseasesBone DiseasesDevelopmental Endocrine System Diseases Natriuretic Peptide, C-Type
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.