RecruitingPhase 2

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

United States100 participantsStarted 2024-10-21

Plain-language 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).

Who can participate

Age range3 Years – 11 Years

SexALL

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Inclusion criteria

✓. 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
✓. 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).

Exclusion criteria

✕. 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.
✕. Previous treatment with a growth promoting agent

What they're measuring

Change from baseline in Annualized Growth Velocity (AGV)

Timeframe: At 6 months

Change from baseline in height

Timeframe: At 4 years

Change from baseline in height Z-score

Timeframe: At 4 years

Trial details

NCT IDNCT06382155

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-06

Contact for this trial

Trial Specialist

+1 415.475.5852 medinfo@bmrn.com

View on ClinicalTrials.gov More Idiopathic Short Stature trials