A Phase 2 interventional study of BMN 111 in Achondroplasia, sponsored by BioMarin Pharmaceutical. Active, not recruiting at 9 sites in 4 countries. Open to participants aged 7 Years and older. Per ClinicalTrials.gov, last updated 2026-03-13.
Sponsored by BioMarin Pharmaceutical · Phase 2, Interventional, and Treatment
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
47 studies on the registry are indexed under Achondroplasia; 13 are open to participants now.
This study's enrollment of 30 is below 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.
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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.
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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.
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.
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.
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.
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.
111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.
Drug: BMN 111
BMN 111 will be administered subcutaneously daily.
Also known as: Modified recombinant human C-type natriuretic peptide, Vosoritide
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Growth Velocity [Efficacy]
Annualized growth velocity (cm/yr)
Time frame: Until near final adult height is reached
Growth Parameters (Efficacy)
Height standard score (Z-score)
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Body Proportions (Efficacy)
Upper-to-lower body segment ratio
Time frame: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later
Final Adult Height
Height at the age of 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
Plan to share: No
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