A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Idiopathic Short Stature Clinical Trial

Official title:

A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06382155
• Other study ID #: 111-210
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: September 2024
• Est. completion date: December 2036

Study information

• Verified date: April 2024
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Not yet recruiting
• Enrollment: 100
• Est. completion date: December 2036
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 10 Years

Eligibility

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. Tanner Stage 1, at time of signing the ICF (unless too young to stage).

Key Exclusions:

1. 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)

2. Previous treatment with a growth promoting agent

Related Conditions & MeSH terms

• Dwarfism
• Idiopathic Short Stature

Intervention

Drug:
• Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
• Human Growth Hormone
Commercial product containing somatotropin
• Placebo
Lyophilized powder for reconstitution

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in Annualized Growth Velocity (AGV)		At 6 months
Primary	Change from baseline in height		At 4 years
Primary	Change from baseline in height Z-score		At 4 years
Secondary	Incidence of treatment-emergent adverse events		Until the end of the study, up to 15 years
Secondary	Change from baseline in AGV Z-score (average stature reference)		At 6 months
Secondary	Change from baseline in height		Every 6 months through the end of study, up to 15 years
Secondary	Change from baseline in height Z score		Every 6 months through the end of study, up to 15 years
Secondary	Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)		Every 6 months through the end of study, up to 15 years
Secondary	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
Secondary	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
Secondary	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
Secondary	Change from baseline in lumbar spine BMD Z-score		Every 6 months through the end of study, up to 15 years
Secondary	Change from baseline in total body (less head) bone mineral content (BMC)		Every 6 months through the end of study, up to 15 years
Secondary	Change from baseline in lumbar spine BMC		Every 6 months through the end of study, up to 15 years
Secondary	Maximum concentration (Cmax) of vosoritide in plasma		Every 6 months through the end of study, up to 15 years
Secondary	Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-8)		Every 6 months through the end of study, up to 15 years
Secondary	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
Secondary	Elimination half-life of vosoritide (t½)		Every 6 months through the end of study, up to 15 years
Secondary	Apparent clearance of vosoritide		Every 6 months through the end of study, up to 15 years
Secondary	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
Secondary	Time vosoritide is present at maximum concentration (Tmax)		Every 6 months through the end of study, up to 15 years