Actively Recruiting
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months
Led by BioMarin Pharmaceutical · Updated on 2026-03-31
60
Participants Needed
26
Research Sites
N/A
Total Duration
On this page
Sponsors
B
BioMarin Pharmaceutical
Lead Sponsor
I
ICON Clinical Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effects of daily vosoritide treatment compared to placebo in infants and young children with hypochondroplasia (HCH), aged from birth up to less than 36 months. This Phase 2, randomized, double-blind, placebo-controlled, multicenter study aims to better understand vosoritide's impact on growth and health over one year in this specific young population with genetically confirmed HCH. Participants will be randomly assigned to receive either vosoritide or a placebo through daily subcutaneous injections for 52 weeks. The vosoritide dose will be adjusted based on weight bands approved for a related condition. After treatment, there will be a 2-week safety follow-up to monitor any adverse effects or changes. During the study, participants will have regular evaluations including clinical lab tests, heart rate, respiratory rate, blood pressure, and body temperature measures at several time points. Researchers will assess growth changes such as height, body segment ratios, and bone density using scans, along with monitoring adverse events and vosoritide blood levels. The total participation duration is about 54 weeks including treatment and safety follow-up.
CONDITIONS
Brief 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 of age at randomization.
- Confirmed genetic diagnosis of hypochondroplasia by whole genome sequencing showing a FGFR3 variant.
- Participants aged 0 to less than 12 months must have a height Z-score of ≤ -1.0 SDS; those aged 12 to less than 36 months must have a height Z-score of ≤ -2.0 SDS based on CDC growth charts.
- Participant's weight at Day 1 visit must be at least 3 kg.
You will not qualify if you...
- Presence of short stature condition other than hypochondroplasia (e.g., achondroplasia, trisomy 21, pseudoachondroplasia).
- Unstable medical condition likely to require surgery during the study period.
- Use of prohibited medications.
- Treatment with growth hormone, IGF-1, or anabolic steroids in past 6 months or long-term treatment over 3 months at any time.
- Need for investigational agents before study completion.
- Receipt of other investigational products or devices within 30 days before screening.
- Previous use of investigational treatments for hypochondroplasia or short stature.
- Current or history of malignancy or under evaluation for suspected malignancy.
- Known hypersensitivity to vosoritide or its ingredients.
- Conditions that may risk poor compliance or study completion.
- Any concurrent disease or condition that may interfere with participation or safety assessments.
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.
Duration - 52 weeks
Participants receive daily subcutaneous injections of vosoritide or placebo based on their weight using a weight-band dosing regimen.
Daily injections with periodic assessments at Week 13, Week 26, Week 39, and Week 52
Duration - 2 weeks
Participants have a safety follow-up visit after completing treatment to monitor any adverse effects.
1 safety follow-up visit (in-person)
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
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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