Vosoritide for Idiopathic Short Stature
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. However, if you have previously used a growth-promoting agent, you may not be eligible to participate.
What data supports the effectiveness of the drug Vosoritide (Voxzogo) for idiopathic short stature?
Vosoritide has been shown to significantly increase growth velocity in children with achondroplasia, a condition that also causes short stature. This suggests it may be effective for other types of short stature, like idiopathic short stature, although direct evidence for this specific use is not provided.12345
Is vosoritide safe for humans?
Vosoritide, also known as Voxzogo, has been approved for use in children with achondroplasia and is generally considered safe, but it can cause a decrease in blood pressure, which is a serious side effect. This risk is outweighed by the benefits for those with achondroplasia, according to regulatory agencies.12467
How is the drug vosoritide unique for treating idiopathic short stature?
Vosoritide is unique because it is a daily injectable drug originally developed to treat achondroplasia, a specific form of dwarfism, by targeting a genetic mutation that affects bone growth. It works by restoring normal bone growth through a mechanism that inhibits the overactive signaling pathways caused by this mutation.12689
What is the purpose of this trial?
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).
Research Team
Medical Director MD
Principal Investigator
BioMarin Pharmaceutical
Eligibility Criteria
This trial is for children with idiopathic short stature, which means they are significantly shorter than their peers without a known cause. They should have a height Z-score ≤ -2.25 and may have had previous growth treatments. Children with certain genetic conditions causing short stature or those in later stages of puberty are not eligible.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Observational
Baseline growth is assessed during a minimum 6-month observational period
Randomized Treatment
Participants receive randomized treatment with vosoritide or placebo for a minimum of 6 months
Open-label Treatment
Participants receive open-label vosoritide until near-final adult height or age threshold is reached
Human Growth Hormone Treatment
Participants in the hGH group receive open-label hGH for a minimum of 4 years
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Vosoritide
Vosoritide is already approved in United States for the following indications:
- To increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses
- to increase linear growth in pediatric patients less than 5 years of age with achondroplasia with open epiphyses
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Who Is Running the Clinical Trial?
BioMarin Pharmaceutical
Lead Sponsor
Alexander Hardy
BioMarin Pharmaceutical
Chief Executive Officer since 2023
MBA from INSEAD
Greg Friberg
BioMarin Pharmaceutical
Chief Medical Officer
MD from New York Medical College