NCT03989947

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

A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 73 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 15 Months
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.	Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)

Timeline

• Start date: 2019-06-12
• Primary completion: 2038-05-01
• Completion: 2038-05-01
• First posted: 2019-06-18
• Last updated: 2026-03-13

Locations

16 sites across 4 countries: United States, Australia, Japan, United Kingdom

Regulatory

• FDA-regulated drug study