A Phase 2 Study of Vosoritide in Children with Idiopathic Short Stature
NCT ID: NCT06382155
Last Updated: 2025-01-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
100 participants
INTERVENTIONAL
2024-10-21
2036-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Vosoritide Dose 1 - Low Dose
Vosoritide Dose 1 daily injection
Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Vosoritide Dose 2 - Medium Dose
Vosoritide Dose 2 daily injection
Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Vosoritide Dose 3 - High Dose
Vosoritide Dose 3 daily injection
Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Placebo
Placebo daily injection
Placebo
Lyophilized powder for reconstitution
Human Growth Hormone
hGH daily injection
Human Growth Hormone
Commercial product containing somatotropin
Interventions
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Vosoritide Injection
Experimental Drug Lyophilized powder for reconstitution
Human Growth Hormone
Commercial product containing somatotropin
Placebo
Lyophilized powder for reconstitution
Eligibility Criteria
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Inclusion Criteria
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
3 Years
10 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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Center Of Excellence in Diabetes and Endocrinology
Sacramento, California, United States
Lundquist Institute for Biomedical Innovation (LA BioMed)
Torrance, California, United States
Nemours Children's Health System - Corporate Headquarters
Pensacola, Florida, United States
Centricity Research
Columbus, Georgia, United States
St. Luke's Children's Endocrinology
Boise, Idaho, United States
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, Idaho, United States
UBMD Pediatrics
Buffalo, New York, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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111-210
Identifier Type: -
Identifier Source: org_study_id
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