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Vosoritide for Idiopathic Short Stature
Phase 2
Recruiting
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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
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)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Summary
"This trial aims to see how well vosoritide works in children with short stature compared to human growth hormone."
Who is the study for?
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.
What is being tested?
The study tests the effects of vosoritide injections compared to human growth hormone in children with ISS. Vosoritide's potential to promote growth will be evaluated over multiple doses and against the established treatment of human growth hormone.
What are the potential side effects?
Possible side effects from vosoritide could include reactions at the injection site, headache, joint pain, and nausea. Human Growth Hormone might cause swelling, joint pain, muscle pain, and changes in glucose tolerance.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My height is significantly below average for my age and sex.
Select...
I have a genetic condition causing short stature, such as Turner syndrome or Noonan syndrome.
Select...
I have been treated with a drug that stimulates growth before.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
Trial Design
5Treatment groups
Experimental Treatment
Active Control
Placebo Group
Group I: Vosoritide Dose 3 - High DoseExperimental Treatment1 Intervention
Vosoritide Dose 3 daily injection
Group II: Vosoritide Dose 2 - Medium DoseExperimental Treatment1 Intervention
Vosoritide Dose 2 daily injection
Group III: Vosoritide Dose 1 - Low DoseExperimental Treatment1 Intervention
Vosoritide Dose 1 daily injection
Group IV: Human Growth HormoneActive Control1 Intervention
hGH daily injection
Group V: PlaceboPlacebo Group1 Intervention
Placebo daily injection
Find a Location
Who is running the clinical trial?
BioMarin PharmaceuticalLead Sponsor
160 Previous Clinical Trials
114,962 Total Patients Enrolled
Medical Director MDStudy DirectorBioMarin Pharmaceutical
1 Previous Clinical Trials
73 Total Patients Enrolled