A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

Clinical Trial

Offered by: Nemours Children's
Location: Delaware Valley

Trial Name

A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

What is the trial about?

This research is being done to learn if a study drug called vosoritide can help children with Turner syndrome, SHOX deficiency or Noonan syndrome who have short stature to grow. The study will compare the effects, good or bad, of vosoritide and human growth hormone on your child’s short stature. New things are learned in research studies like this one by doing tests and giving experimental drugs to people to see what will happen.

Who can participate?

Eligible participants will be aged ≥ 3 years and < 10 years (females) or < 11 years (males) and have a height Z-score of ≤ -2.00 standard deviations (SDs). They must have been receiving continuous treatment with hGH for ≥ 1 year, and be receiving a dose of ≥ 0.35 mg/kg weekly with no dose changes in the last 6 months and no future dose changes planned. Additionally, we will need to complete a screening call confirming the eligibility criteria. A screening visit will occur around 6 months before the administration of study treatment (vosoritide or HGH).

What is involved?

Whether your child is in one of the groups that receives vosoritide, or switches to vosoritide from human growth hormone (after at least 2 years), they may continue study treatment until they stop growing and remain in the study until around ages 16 years for females and 18 years for males. Participants will visit the study site on the first and second day after treatment initiation. Following this, all participants will visit the study site every 3 months for the first year, then 6-monthly thereafter. Other visits will be conducted remotely via telephone unless a site visit is required by the participant or investigator. 

Blood samples will be collected from your child at various time points during the study. The total amount of blood collected will be approximately 7 tablespoons (105 mL) in the first year of the study, and then approximately 3 tablespoons (45 mL) in each year of the study thereafter. Some of the test to be conducted are similar to the Screening tests and procedures such as anthropometric measurements, clinical hip assessment, physical examination, tanner stage of pubertal development, menstrual history in female participants (if applicable), pregnancy test (if applicable), clinical laboratory tests, measurement of vitamin D, IGF-1 test, DXA scan, X-rays, ECG, ECHO scan, ECG trace, questionnaires, current medications and procedures, adverse events, etc.

Please feel free to contact Biomarin@nemours.org for more information about the study.