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An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

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NCT03989947 · readout ≤ 4,303 d

Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.

Phase
Phase 2
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
73actual
Sites
16
Countries
Australia, Japan, United Kingdom +1

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis
DateFiledBasisWhat it is
Start2019-06-12actualWhen the study began enrolling
Primary completionMay 2038estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completionMay 2038estimatedThe whole study's end, after follow-up
First posted2019-06-18actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-03-13actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Achondroplasia

Intervention

  • Drug: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. — also filed as Vosoritide

Primary outcomes

what the primary-completion date above is the date OF
Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
measured "Through study completion, an average of 5 years"
Evaluate change in height/length z-score in children with ACH treated with BMN 111
measured "Through study completion, an average of 5 years"

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