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

Phase 2RecruitingNCT06382155

BioMarin Pharmaceutical100 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

100

Conditions

Idiopathic Short Stature

Interventions

Vosoritide InjectionDRUG

Experimental Drug Lyophilized powder for reconstitution

Human Growth HormoneDRUG

Commercial product containing somatotropin

PlaceboDRUG

Lyophilized powder for reconstitution

Eligibility

Sex: ALLMin age: 3 YearsMax age: 10 Years

Medical Language ↔ Plain English

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

Locations (8)

Center Of Excellence in Diabetes and Endocrinology

Sacramento, California, United States

RECRUITING

Lundquist Institute for Biomedical Innovation (LA BioMed)

Torrance, California, United States

RECRUITING

Outcomes

Primary Outcomes

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

Incidence of treatment-emergent adverse events

Time frame: Until the end of the study, up to 15 years

Change from baseline in AGV Z-score (average stature reference)

Time frame: At 6 months

Change from baseline in height