Trials / Recruiting
RecruitingNCT06668805
A 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
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.
- Status
- Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 54 (estimated)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- All
- Age
- 3 Years – 11 Years
- Healthy volunteers
- Not accepted
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.
Detailed 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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Vosoritide Injection | Modified recombinant human C-type natriuretic peptide Vosoritide |
Timeline
- Start date
- 2024-11-22
- Primary completion
- 2027-03-01
- Completion
- 2041-09-01
- First posted
- 2024-10-31
- Last updated
- 2026-02-27
Locations
24 sites across 5 countries: United States, Australia, France, Italy, Spain
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06668805. Inclusion in this directory is not an endorsement.