A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
- Conditions
- Idiopathic Short Stature
- Interventions
- Registration Number
- NCT06382155
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief 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).
- Detailed 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- 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).
Key Exclusions:
- Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome)
- Previous treatment with a growth promoting agent
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Vosoritide Dose 3 - High Dose Vosoritide Injection Vosoritide Dose 3 daily injection Vosoritide Dose 2 - Medium Dose Vosoritide Injection Vosoritide Dose 2 daily injection Vosoritide Dose 1 - Low Dose Vosoritide Injection Vosoritide Dose 1 daily injection Placebo Placebo Placebo daily injection Human Growth Hormone Human Growth Hormone hGH daily injection
- Primary Outcome Measures
Name Time Method Change from baseline in height At 4 years Change from baseline in Annualized Growth Velocity (AGV) At 6 months Change from baseline in height Z-score At 4 years
- Secondary Outcome Measures
Name Time Method Change from baseline in total body (less head) bone mineral content (BMC) Every 6 months through the end of study, up to 15 years Change from baseline in lumbar spine BMC Every 6 months through the end of study, up to 15 years Maximum concentration (Cmax) of vosoritide in plasma Every 6 months through the end of study, up to 15 years Change from baseline in AGV Z-score (average stature reference) At 6 months Change from baseline in height Every 6 months through the end of study, up to 15 years Change from baseline in total body (less head) bone mineral density (BMD) Z-score Every 6 months through the end of study, up to 15 years Apparent clearance of vosoritide Every 6 months through the end of study, up to 15 years Change from baseline at pre-specified timepoints in serum collagen X marker (CXM) Every 6 months through the end of study, up to 15 years Change from baseline in height Z score Every 6 months through the end of study, up to 15 years Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP) Every 6 months through the end of study, up to 15 years Change from baseline in lumbar spine BMD Z-score Every 6 months through the end of study, up to 15 years Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F) Every 6 months through the end of study, up to 15 years Incidence of treatment-emergent adverse events Until the end of the study, up to 15 years Change from baseline in bone age minus chronological age at pre-specified timepoints Every 6 months through the end of study, up to 15 years Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞) Every 6 months through the end of study, up to 15 years Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t) Every 6 months through the end of study, up to 15 years Elimination half-life of vosoritide (t½) Every 6 months through the end of study, up to 15 years Time vosoritide is present at maximum concentration (Tmax) Every 6 months through the end of study, up to 15 years
Trial Locations
- Locations (8)
Centricity Research
🇺🇸Columbus, Georgia, United States
Nemours Children's Health System - Corporate Headquarters
🇺🇸Pensacola, Florida, United States
Rocky Mountain Clinical Research - Idaho Falls
🇺🇸Idaho Falls, Idaho, United States
Center Of Excellence in Diabetes and Endocrinology
🇺🇸Sacramento, California, United States
Lundquist Institute for Biomedical Innovation (LA BioMed)
🇺🇸Torrance, California, United States
UBMD Pediatrics
🇺🇸Buffalo, New York, United States
Children's Hospital at Montefiore
🇺🇸Bronx, New York, United States
St. Luke's Children's Endocrinology
🇺🇸Boise, Idaho, United States