A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
BioMarin Pharmaceutical
Summary
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).
Description
Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years. A Treatment Completion visit will occur approximately 4 weeks after the last ad…
Eligibility
- Age range
- 3–11 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts 2. If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs). Key Exclusions: 1. Known chromosomal imbalance or genetic variant causing shor…
Interventions
- DrugVosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
- DrugHuman Growth Hormone
Commercial product containing somatotropin
- DrugPlacebo
Lyophilized powder for reconstitution
Locations (47)
- Consano Clinical Research, LLCCalifornia City, California
- Rady Children's Hospital - San DiegoCalifornia City, California
- Children's Hospital of Orange County Main Campus - OrangeCalifornia City, California
- Children's Hospital Los AngelesLos Angeles, California
- Children's Hospital Oakland Research InstituteOakland, California
- Center Of Excellence in Diabetes and EndocrinologySacramento, California