A Phase 3 interventional study of BMN 111 in Achondroplasia, sponsored by BioMarin Pharmaceutical. Active, not recruiting at 24 sites in 7 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-03-13.
Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
47 studies on the registry are indexed under Achondroplasia; 13 are open to participants now.
This study's enrollment of 119 is above the median of 74 across 28 interventional studies indexed under Achondroplasia.
Browse Achondroplasia studies →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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
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.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: BMN 111
Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Also known as: Vosoritide, Modified recombinant human C-type natriuretic peptide
Change from baselines in mean annualized growth velocity
Long term efficacy as measured by change in annualized growth velocity
Time frame: Through study completion, an average of 1 year
Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire
Time frame: Through study completion, every 6-12 months
Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire
Time frame: Through study completion, every 12 months
Characterize maximum concentration (Cmax) of BMN 111 in plasma
Time frame: Through study completion, every 12 months
Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame: Through study completion, every 12 months
Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time frame: Through study completion, every 12 months
Characterize the elimination half-life of BMN 111 (t1⁄2)
Time frame: Through study completion, every 12 months
Characterize the apparent clearance of drug
Time frame: Through study completion, every 12 months
Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)
Time frame: Through study completion, every 12 months
Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)
Time frame: Through study completion, every 12 months
BMN 111 Activity Biomarkers
BMN 111 activity will be assessed by measuring bone and collagen metabolism
Time frame: Through study completion, every 12 months
Evaluate change from baseline in body proportion ratios of the extremities
Time frame: Through study completion, every 6 months
Effect of BMN 111 on bone morphology and quality
The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via X-Ray and Dual X-ray Absorptiometry
Time frame: Through study completion, every 12 months for DXA or 2 years for X-ray
Final Adult Height
Height at 16 years for females and 18 years for males
Time frame: Up to at least 16 years of age for females and 18 years of age for males
Optional exploratory genomic biomarker analysis
Exploratory genomic analysis of genes associated with CNP signaling
Time frame: Once through study completion
Plan to share: No
Started Dec 2017 (actual). Primary completion is estimated for Jun 2031, 162 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Vosoritide