A Phase 2 interventional study of Vosoritide and Placebo in Hypochondroplasia, sponsored by BioMarin Pharmaceutical. Recruiting at 26 sites in 7 countries. Open to participants aged 0 Months to 36 Months. Per ClinicalTrials.gov, last updated 2026-03-31.
Sponsored by BioMarin Pharmaceutical · Phase 2, Interventional, and Treatment
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.
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.
BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Drug: Vosoritide
Subcutaneous injection of recommended dose of placebo
Drug: Placebo
The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
Subcutaneous injection of recommended dose of placebo
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
Units of measure: bpm
Time frame: At week 13, at week 26, at week 39, at week 52
Change from baseline in height Z-score
Time frame: At week 52
Changes from baseline in respiratory rate
Units of measure: breaths/min
Time frame: At week 13, at week 26, at week 39, at week 52
Changes from baseline in temperature
Units of measure: celsius
Time frame: At week 13, at week 26, at week 39, at week 52
Changes from baseline in blood pressure
Units of measure: mmHg
Time frame: At week 13, at week 26, at week 39, at week 52
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
Plan to share: No
No publications or documents are linked to this record.
Started Jul 2025 (actual). Primary completion is estimated for Jun 2028, 35 months after the start.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Vosoritide
Hypochondroplasia