A Phase 2, Randomized, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment.
BioMarin Pharmaceutical
Summary
The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.
Description
This is a Phase 2, randomized, multicenter, basket study of vosoritide in children with Turner syndrome, short stature homeobox-containing gene (SHOX) deficiency, or Noonan syndrome who have inadequate growth during or after human growth hormone (hGH) treatment. The study is intended to characterize the short-term efficacy and safety of 3 dosing regimens of vosoritide. The efficacy and safety of the vosoritide therapeutic dose will be further evaluated, and an analysis of the impact of vosoritide on final adult height (FAH).
Eligibility
- Age range
- 3–11 years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- DrugVosoritide Injection
Modified recombinant human C-type natriuretic peptide Vosoritide
Locations (24)
- Children's Hospital of Orange County Main Campus - OrangeOrange, California
- Children's Hospital ColoradoAurora, Colorado
- Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)Wilmington, Delaware
- Children's National Medical CenterWashington D.C., District of Columbia
- Nicklaus Children's HospitalMiami, Florida
- St. Luke's Children's Endocrinology and DiabetesBoise, Idaho