HypochondroplasiaPhase 2NCT07126262
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
A Phase 2 study testing Vosoritide in people with Hypochondroplasia, run by BioMarin Pharmaceutical at 26 sites in 7 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 0 to 36 months
- Conditions the study is for
- Hypochondroplasia
- What is being tested?
- Vosoritide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 26 sitesin 7 countries
- Study dates
- 30 Jul 2025to 30 Jun 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants must be 0 to < 36 months of age at randomization.
- Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
- 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.
- Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
Who cannot join
- Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
- Have an unstable medical condition likely to require surgical intervention during the study period.
- Taking any of the prohibited medications.
- Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time.
- Require any investigational agent prior to completion of study period.
- Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
Show all 11Show fewer
- Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
- Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
- Have known hypersensitivity to vosoritide or its excipients.
- Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.
- Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.
Trial locations and contacts
26 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
26 sites in 7 countries
United States10 sites, 10 recruiting
- Recruiting
The Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
- Recruiting
University of Missouri
Columbia, Missouri, 65211
- Not yet recruiting
Cook Children's Endocrinology
Fort Worth, Texas, 76104
- Not yet recruiting
Cedars-Sinai Medical Center
Los Angeles, California, 90048
- Recruiting
University of Minneasota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
- Not yet recruiting
Children's Wisconsin - Fox Valley Hospital
Neenah, Wisconsin, 54956
- Recruiting
Benioff Children's Hospital - Oakland
Oakland, California, 94609
- Recruiting
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix, Arizona, 85016
- Recruiting
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Japan5 sites, 5 recruiting
- Recruiting
Kumamoto University Hospital
Kumamoto, 860-8556
- Recruiting
Osaka Women's and Children's Hospital
Osaka, 94-1101
- Recruiting
Institute of Science Tokyo Hospital
Tokyo, 113-8519
- Recruiting
Nihon University Itabashi Hospital
Tokyo, 173-8610
- Recruiting
Tottori University Hospital
Tottori, 683-0854
Germany3 sites, 3 recruiting
- Recruiting
Uniklinik Köln
Cologne, North Rhine-Westphalia, 50937
- Recruiting
Universitätsklinikum des Saarlandes
Homburg, Saarland, 66421
- Not yet recruiting
Universitätskinderklinik Magdeburg
Magdeburg, Saxony-Anhalt, 39120
Australia2 sites, 2 recruiting
- Recruiting
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
- Not yet recruiting
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, 4101
France2 sites, 2 recruiting
- Not yet recruiting
Hospices Civils de Lyon - Hôpital Femme Mère Enfant
Bron, Rhone-Alps, 69500
- Recruiting
Hôpital Bicêtre
Le Kremlin-Bicêtre, Paris, 94270
Italy2 sites, 2 recruiting
- Recruiting
IRCCS Istituto Giannina Gaslini
Genova, 16147
- Not yet recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Rome, 00168
United Kingdom2 sites, 2 recruiting
- Not yet recruiting
Myriad Trials
London, England, W1W 5DT
- Not yet recruiting
Great Ormond Street Hospital
London, England, WC1N3BH
About this study
Official title: 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
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.
Read the detailed description
Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH.
Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.
- Conditions
- Hypochondroplasia
- Being tested
- Vosoritide, Placebo
- Medical subject headings
- Hypochondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VosoritideDrug
The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
PlaceboDrug
Subcutaneous injection of recommended dose of placebo
| Study group | Role | What they receive |
|---|---|---|
| Vosoritide injection with vial and syringe | Experimental | Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily. Receives: Drug: Vosoritide |
| Placebo injection with vial and syringe | Placebo comparator | Subcutaneous injection of recommended dose of placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events
Measured: From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
Measured: From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Measured: At week 26, at week 52
Changes from baseline in heart rate
Units of measure: bpm
Measured: At week 13, at week 26, at week 39, at week 52
Change from baseline in height Z-score
Measured: At week 52
Changes from baseline in respiratory rate
Units of measure: breaths/min
Measured: At week 13, at week 26, at week 39, at week 52
Changes from baseline in temperature
Units of measure: celsius
Measured: At week 13, at week 26, at week 39, at week 52
Changes from baseline in blood pressure
Units of measure: mmHg
Measured: At week 13, at week 26, at week 39, at week 52
Secondary outcomes (19)
Change in height
Measured: At week 52
Cumulative annualized growth velocity (AGV)
Measured: At week 52
6-month interval AGV
Measured: At week 26, at week 52
Change from baseline in upper to lower body segment ratio
Measured: At week 52
Change from baseline in arm span
Measured: At week 52
Change from baseline in total body (less head) bone mineral density (BMD) Z-score
Measured: At week 52
Change from baseline in lumbar spine BMD Z-score
Measured: At week 52
Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA
Measured: At week 52
Change from baseline in lumbar spine BMC as measured by DXA
Measured: At week 52
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Measured: At week 26, at week 52
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Measured: At week 26, at week 52
Elimination half-life of vosoritide (t½)
Measured: At week 26, at week 52
Apparent clearance of vosoritide
Measured: At week 26, at week 52
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Measured: At week 26, at week 52
Time vosoritide is present at maximum concentration (Tmax)
Measured: At week 26, at week 52
Maximum concentration (Cmax) of vosoritide in plasma
Measured: At week 26, at week 52
Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)
Measured: At week 26 and week 52
Incidence of otitis media
Measured: From baseline to end of treatment at 52 weeks
Seizure frequency over the course of the study
Measured: From baseline to end of treatment at 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2025
- Study start30 Jul 2025
- Primary completion30 Jun 2028 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypochondroplasiaStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON Clinical Research
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07126262
- Sponsor's protocol ID
- 111-212
- Status last verified
- Mar 2026
- First submitted
- 29 Jul 2025
- Last updated
- 31 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07126262
- Acronym / sponsor ID
- 111-212
- Conditions
- Hypochondroplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Collaborators
- ICON Clinical Research
- Study start
- 30 Jul 2025 (actual)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 17 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH.
Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Vosoritide injection with vial and syringe | Experimental | Drug: VosoritideSubcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily. |
| Placebo injection with vial and syringe | Placebo comparator | Drug: PlaceboSubcutaneous injection of recommended dose of placebo |
Who may qualify
- Ages
- 0 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Participants must be 0 to < 36 months of age at randomization.
- Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
- 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.
- Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
Key exclusion criteria (11 listed)
- Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
- Have an unstable medical condition likely to require surgical intervention during the study period.
- Taking any of the prohibited medications.
- Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time.
- Require any investigational agent prior to completion of study period.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VosoritideThe vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH | Drug | Vosoritide injection with vial and syringe |
| PlaceboSubcutaneous injection of recommended dose of placebo | Drug | Placebo injection with vial and syringe |
Primary outcome
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
19 secondary outcomes
Change in height
Time frame: At week 52
Cumulative annualized growth velocity (AGV)
Time frame: At week 52
6-month interval AGV
Time frame: At week 26, at week 52
Change from baseline in upper to lower body segment ratio
Time frame: At week 52
Change from baseline in arm span
Time frame: At week 52
Change from baseline in total body (less head) bone mineral density (BMD) Z-score
Time frame: At week 52
Change from baseline in lumbar spine BMD Z-score
Time frame: At week 52
Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA
Time frame: At week 52
Change from baseline in lumbar spine BMC as measured by DXA
Time frame: At week 52
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame: At week 26, at week 52
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time frame: At week 26, at week 52
Elimination half-life of vosoritide (t½)
Time frame: At week 26, at week 52
Apparent clearance of vosoritide
Time frame: At week 26, at week 52
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Time frame: At week 26, at week 52
Time vosoritide is present at maximum concentration (Tmax)
Time frame: At week 26, at week 52
Maximum concentration (Cmax) of vosoritide in plasma
Time frame: At week 26, at week 52
Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)
Time frame: At week 26 and week 52
Incidence of otitis media
Time frame: From baseline to end of treatment at 52 weeks
Seizure frequency over the course of the study
Time frame: From baseline to end of treatment at 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
26 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vosoritide in people with Hypochondroplasia, run by BioMarin Pharmaceutical at 26 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Japan, Germany, Australia, France and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Specialist (1-800-983-4587, medinfo@bmrn.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (time frame: From baseline to end of treatment at 52 weeks). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2026.