A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children with Idiopathic Short Stature
Status: Recruiting
Location: See all (8) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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), in children with idiopathic short stature (ISS).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 10
Healthy Volunteers: f
View:
• 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
• Tanner Stage 1, at time of signing the ICF (unless too young to stage).
Locations
United States
California
Center Of Excellence in Diabetes and Endocrinology
RECRUITING
Sacramento
Lundquist Institute for Biomedical Innovation (LA BioMed)
RECRUITING
Torrance
Florida
Nemours Children's Health System - Corporate Headquarters
RECRUITING
Pensacola
Georgia
Centricity Research
RECRUITING
Columbus
Idaho
St. Luke's Children's Endocrinology
RECRUITING
Boise
Rocky Mountain Clinical Research - Idaho Falls
RECRUITING
Idaho Falls
New York
UBMD Pediatrics
RECRUITING
Buffalo
Children's Hospital at Montefiore
RECRUITING
The Bronx
Contact Information
Primary
Trial Specialist
medinfo@bmrn.com
+1 415.475.5852
Time Frame
Start Date: 2024-10-21
Estimated Completion Date: 2036-12
Participants
Target number of participants: 100
Treatments
Experimental: Vosoritide Dose 1 - Low Dose
Vosoritide Dose 1 daily injection
Experimental: Vosoritide Dose 2 - Medium Dose
Vosoritide Dose 2 daily injection
Experimental: Vosoritide Dose 3 - High Dose
Vosoritide Dose 3 daily injection
Placebo_comparator: Placebo
Placebo daily injection
Active_comparator: Human Growth Hormone
hGH daily injection
Related Therapeutic Areas
Sponsors
Leads: BioMarin Pharmaceutical