Actively Recruiting
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Led by BioMarin Pharmaceutical · Updated on 2026-03-31
60
Participants Needed
26
Research Sites
152 weeks
Total Duration
On this page
Sponsors
B
BioMarin Pharmaceutical
Lead Sponsor
I
ICON Clinical Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
CONDITIONS
Official Title
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 0 to less than 36 months old at randomization
- Confirmed genetic diagnosis of hypochondroplasia via whole genome sequencing showing FGFR3 pathogenic variant
- Participants aged 0 to less than 12 months must have a height Z-score of 64; -1.0 SDS
- Participants aged 12 to less than 36 months must have a height Z-score of 64; -2.0 SDS
- Participant's weight at Day 1 visit must be at least 3 kilograms
You will not qualify if you...
- Short stature conditions other than hypochondroplasia (e.g., achondroplasia, trisomy 21, pseudoachondroplasia)
- Unstable medical conditions likely requiring surgical intervention during the study
- Taking any prohibited medications
- Treated with growth hormone, IGF-1, or anabolic steroids in the 6 months prior to screening or long-term treatment (> 3 months) at any time
- Need for any investigational agent before study completion
- Received another investigational product or device within 30 days prior to screening
- Used investigational products or devices for hypochondroplasia or short stature treatment at any time
- Current or history of malignancy, or under evaluation for suspected malignancy
- Known hypersensitivity to vosoritide or its ingredients
- Conditions or circumstances that increase risk for poor treatment adherence or study completion
- Concurrent diseases or conditions interfering with participation or safety assessments
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 26 locations
1
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
Not Yet Recruiting
3
Benioff Children's Hospital - Oakland
Oakland, California, United States, 94609
Actively Recruiting
4
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
5
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
6
The Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
Actively Recruiting
7
University of Minneasota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
8
University of Missouri
Columbia, Missouri, United States, 65211
Actively Recruiting
9
Cook Children's Endocrinology
Fort Worth, Texas, United States, 76104
Not Yet Recruiting
10
Children's Wisconsin - Fox Valley Hospital
Neenah, Wisconsin, United States, 54956
Not Yet Recruiting
11
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, Australia, 4101
Not Yet Recruiting
12
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Actively Recruiting
13
Hôpital Bicêtre
Le Kremlin-Bicêtre, Paris, France, 94270
Actively Recruiting
14
Hospices Civils de Lyon - Hôpital Femme Mère Enfant
Bron, Rhone-Alps, France, 69500
Not Yet Recruiting
15
Uniklinik Köln
Cologne, North Rhine-Westphalia, Germany, 50937
Actively Recruiting
16
Universitätsklinikum des Saarlandes
Homburg, Saarland, Germany, 66421
Actively Recruiting
17
Universitätskinderklinik Magdeburg
Magdeburg, Saxony-Anhalt, Germany, 39120
Not Yet Recruiting
18
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Rome, Italy, 00168
Not Yet Recruiting
19
IRCCS Istituto Giannina Gaslini
Genova, Italy, 16147
Actively Recruiting
20
Kumamoto University Hospital
Kumamoto, Japan, 860-8556
Actively Recruiting
21
Osaka Women's and Children's Hospital
Osaka, Japan, 94-1101
Actively Recruiting
22
Institute of Science Tokyo Hospital
Tokyo, Japan, 113-8519
Actively Recruiting
23
Nihon University Itabashi Hospital
Tokyo, Japan, 173-8610
Actively Recruiting
24
Tottori University Hospital
Tottori, Japan, 683-0854
Actively Recruiting
25
Myriad Trials
London, England, United Kingdom, W1W 5DT
Not Yet Recruiting
26
Great Ormond Street Hospital
London, England, United Kingdom, WC1N3BH
Not Yet Recruiting
Research Team
T
Trial Specialist
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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