NCT07126262 · BioMarin Pharmaceutical
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
What this study is about
The purpose of this study is to evaluate the safety and effectiveness of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
View original scientific description
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.
Interventions
DRUG
Vosoritide
The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
DRUG
Placebo
Subcutaneous injection of recommended dose of placebo
Primary outcome measures
Incidence of treatment-emergent adverse events
Time frame: From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
Time frame: From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Time frame: At week 26, at week 52
Changes from baseline in heart rate
Time frame: At week 13, at week 26, at week 39, at week 52
Units of measure: bpm
Change from baseline in height Z-score
Time frame: At week 52
Changes from baseline in respiratory rate
Time frame: At week 13, at week 26, at week 39, at week 52
Units of measure: breaths/min
Changes from baseline in temperature
Time frame: At week 13, at week 26, at week 39, at week 52
Units of measure: celsius
Changes from baseline in blood pressure
Time frame: At week 13, at week 26, at week 39, at week 52
Units of measure: mmHg
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Participants must be 0 to \< 36 months of age at randomization. 2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). 3. Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts. 4. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg. Key
Exclusion criteria
- Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia). 2. Have an unstable medical condition likely to require surgical intervention during the study period. 3. Taking any of the prohibited medications. 4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroi
Where
- Phoenix, Arizona
- Los Angeles, California
- Oakland, California
- Washington D.C., District of Columbia
- Chicago, Illinois
- Baltimore, Maryland
- Minneapolis, Minnesota
- Columbia, Missouri
- Fort Worth, Texas
- Neenah, Wisconsin
Collaborators
ICON Clinical Research
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 31, 2026 · Source of record for eligibility and locations