Actively Recruiting
A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide Compared to Human Growth Hormone in Children With Idiopathic Short Stature
Led by BioMarin Pharmaceutical · Updated on 2026-05-22
100
Participants Needed
47
Research Sites
548 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the effects of different doses of vosoritide and compare the therapeutic dose of vosoritide to human growth hormone (hGH) in children diagnosed with idiopathic short stature (ISS). The study is a Phase 2, randomized, controlled trial that seeks to understand how these treatments influence growth in affected children. Participants will first undergo a minimum 6-month observation period to assess their baseline growth. Then, those assigned to the vosoritide and placebo groups will receive randomized treatment for at least 6 months, with placebo limited to a maximum of 6 months. After this, open-label vosoritide treatment will continue until participants reach near-final adult height or at least 16 years for females and 18 years for males, whichever is later. Participants randomized to the hGH group will receive open-label hGH treatment for a minimum of 4 years. Study treatments involve daily injections. Throughout the study, participants will attend regular visits for clinical and imaging assessments, including evaluations of hips and lower extremities. Researchers will monitor safety concerns such as hypotension, fractures, and slipped capital femoral epiphysis, with oversight from an independent Data Monitoring Committee. The main outcomes measured include changes in annualized growth velocity at 6 months and height changes over 4 years. Follow-up assessments will continue as needed, including safety monitoring, until study completion, which could last up to 15 years.
CONDITIONS
Brief Title
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Child aged 3 to 11 years
- Height Z-score of -2.25 or lower compared to general population for age and sex
- If 5 years or older, must be at Tanner Stage I
- Historic test showing growth hormone level above 10 μg/L or normal IGF-1 levels for age
You will not qualify if you...
- Known genetic or chromosomal conditions causing short stature, such as Laron syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, Noonan syndrome, or ACAN deficiency
- Previous treatment with growth promoting agents
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Minimum 6 months
Participants undergo a minimum 6 month observational period to assess baseline growth before starting treatment.
Visits as scheduled for growth assessments during observation
Duration - Minimum 6 months for vosoritide and placebo groups; up to 4 years for human growth hormone group
Participants receive daily injections of vosoritide, placebo, or human growth hormone according to their assigned group.
Daily injections with regular clinical and imaging assessments during treatment
Duration - Up to approximately 15 years depending on participant age and growth
Participants in the vosoritide groups continue open-label treatment until near-final adult height or specified age; human growth hormone group continues open-label treatment for a minimum of 4 years.
Ongoing treatment with regular clinical and imaging assessments
Duration - Approximately 4 weeks after last dose plus ongoing follow-up up to study end
Participants complete a treatment completion visit approximately 4 weeks after last injection, with follow-up assessments including annual and periodic evaluations through the end of the study.
1 treatment completion visit and annual or periodic follow-up visits
Trial Site Locations
Total: 47 locations
1
Consano Clinical Research, LLC
California City, California, United States, 78231
Actively Recruiting
2
Rady Children's Hospital - San Diego
California City, California, United States, 92123
Actively Recruiting
3
Children's Hospital of Orange County Main Campus - Orange
California City, California, United States, 92868
Actively Recruiting
4
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
5
Children's Hospital Oakland Research Institute
Oakland, California, United States, 94609
Actively Recruiting
6
Center Of Excellence in Diabetes and Endocrinology
Sacramento, California, United States, 95821
Actively Recruiting
7
Lundquist Institute for Biomedical Innovation (LA BioMed)
Torrance, California, United States, 90502
Actively Recruiting
8
The Children's National Research Institute
Washington D.C., District of Columbia, United States, 20012
Actively Recruiting
9
Nemours Children's Specialty Care Jacksonville
Jacksonville, Florida, United States, 32207
Actively Recruiting
10
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
11
Optimal Research Sites
Orange City, Florida, United States, 32763
Actively Recruiting
12
Nemours Children's Health System - Corporate Headquarters
Pensacola, Florida, United States, 32514
Actively Recruiting
13
Centricity Research
Columbus, Georgia, United States, 31904
Actively Recruiting
14
St. Luke's Children's Endocrinology
Boise, Idaho, United States, 83712
Withdrawn
15
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, Idaho, United States, 83404
Actively Recruiting
16
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
Actively Recruiting
17
Kentucky Children's Hospital
Lexington, Kentucky, United States, 40536
Actively Recruiting
18
University of Louisville School of Medicine
Louisville, Kentucky, United States, 40202
Actively Recruiting
19
Velocity Clinical Research - MedPharmics - Lafayette
Lafayette, Louisiana, United States, 70508
Actively Recruiting
20
Ochsner Health Center for Children - New Orleans
New Orleans, Louisiana, United States, 70121
Withdrawn
21
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
22
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
23
UBMD Pediatrics
Buffalo, New York, United States, 14203
Actively Recruiting
24
NYU Langone Ambulatory Care Garden City
Garden City, New York, United States, 11530
Withdrawn
25
Atlantic Health Children's Specialty Center at Morristown
New York, New York, United States, 07960
Actively Recruiting
26
The Clinical and Translational Science Institute
New York, New York, United States, 10016
Actively Recruiting
27
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Actively Recruiting
28
Children's Hospital at Montefiore
The Bronx, New York, United States, 10467
Actively Recruiting
29
University of North Carolina Children's Specialty Clinic
Chapel Hill, North Carolina, United States, 27514
Actively Recruiting
30
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
31
Prisma Health Children's Hospital - Midlands
Columbia, South Carolina, United States, 29203
Actively Recruiting
32
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
33
Cook Children's Endocrinology
Fort Worth, Texas, United States, 76104
Withdrawn
34
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
35
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
36
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Actively Recruiting
37
Centre Hospitalier Universitaire d'Angers
Angers, France, 49 933
Actively Recruiting
38
Hôpital de la Timone - Enfants
Marseille, France, 13005
Actively Recruiting
39
CHU de Toulouse - Hôpital des Enfants
Toulouse, France, 31059
Actively Recruiting
40
Hôpital Bicêtre
Le Kremlin-Bicêtre, Île-de-France Region, France, 94270
Actively Recruiting
41
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany, 66421
Actively Recruiting
42
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
Florence, Florence, Italy, 50139
Actively Recruiting
43
Ospedale Pediatrico Bambino Gesù
Roma, Rome, Italy, 00165
Actively Recruiting
44
IRCCS Istituto Giannina Gaslini
Genova, Italy, 16147
Actively Recruiting
45
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, South Korea, 13620
Actively Recruiting
46
Inha University Hospital
Incheon, South Korea, 22332
Actively Recruiting
47
Chungnam National University Sejong Hospital (CNUSH)
Sejong, South Korea, 30099
Actively Recruiting
Research Team
T
Trial Specialist
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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