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

NCTNCT06382155Other id111-210

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Overview

Status: Recruiting
Phase: Phase 2
Enrollment: 100 participants (estimated)
Sponsor: BioMarin Pharmaceutical · Industry
Design: Randomized · Quadruple masking
Countries: 6 countries
United States, Australia, France, Germany, Italy, South Korea
Start: 21 Oct 2024
Primary completion: Jun 2026 (estimated)
Completion: Dec 2036 (estimated)
First posted: 24 Apr 2024
Last update posted: 22 May 2026
Conditions: Idiopathic Short Stature

Timeline

1 trial · 1 ongoing

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

202520302035

Ph 2
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
NCT06382155
21 Oct 2024 to est. Dec 2036

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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).

Interventions

Vosoritide InjectionDrug
Human Growth HormoneDrug
PlaceboDrug

Arms5

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 (US only)

Primary outcomes3

Outcomes the sponsor listed as primary.

Change from baseline in Annualized Growth Velocity (AGV)
Time frame · At 6 months
Change from baseline in height
Time frame · At 4 years
Change from baseline in height Z-score
Time frame · At 4 years

Secondary outcomes(18)

Incidence of treatment-emergent adverse events
Time frame · Until the end of the study, up to 15 years
Change from baseline in Height Z-score (average stature reference)
Time frame · At 6 months
Change from baseline in height
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline in height Z score
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)
Time frame · 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)
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline in bone age minus chronological age at pre-specified timepoints
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline in whole body (less head) bone mineral density (BMD) Z-score
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMD Z-score
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline in total body (less head) bone mineral content (BMC)
Time frame · Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMC
Time frame · Every 6 months through the end of study, up to 15 years
Maximum concentration (Cmax) of vosoritide in plasma
Time frame · 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-∞)
Time frame · 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)
Time frame · Every 6 months through the end of study, up to 15 years
Elimination half-life of vosoritide (t½)
Time frame · Every 6 months through the end of study, up to 15 years
Apparent clearance of vosoritide
Time frame · 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)
Time frame · Every 6 months through the end of study, up to 15 years
Time vosoritide is present at maximum concentration (Tmax)
Time frame · Every 6 months through the end of study, up to 15 years

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

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Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: from 3 Years up to 11 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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 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), ACAN deficiency. 2. Previous treatment with a growth promoting agent

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