An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
Condition(s) studied
Investigational drug(s) / intervention(s)
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)
Study 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.
Eligibility
Primary outcome measure(s)
- Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] — "Through study completion, an average of 5 years"
Number of study participants with treatment-emergent adverse events or serious adverse events - Evaluate change in height/length z-score in children with ACH treated with BMN 111 — "Through study completion, an average of 5 years"
Trial sites (16)
| Facility | City | Region | Status |
|---|---|---|---|
| Children's Hospital & Research Center Oakland | Oakland | California | |
| Harbor - UCLA Medical Center | Torrance | California | |
| Alfred I. duPont Hospital for Children | Wilmington | Delaware | |
| Emory University | Decatur | Georgia | |
| Ann Robert and H. Lurie Children's Hospital of Chicago | Chicago | Illinois | |
| Cincinnati Childrens Hospital | Cincinnati | Ohio | |
| Vanderbilt University Medical Center | Nashville | Tennessee | |
| Baylor College of Medicine | Houston | Texas | |
| Medical College of Wisconsin, Children's Hospital | Milwaukee | Wisconsin | |
| The Children's Hospital at Westmead | Westmead | New South Wales | |
| Murdoch Children's Research Institute | Parkville | Victoria | |
| Osaka University | Osaka | Japan | |
| Saitama Children's Medical Center | Saitama | Japan | |
| Tokushima University Hospital | Tokushima | Japan | |
| Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital | London | United Kingdom | |
| Sheffield Children's NHS Foundation Trust | Sheffield | United Kingdom |
More BioMarin Pharmaceutical trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03989947 on ClinicalTrials.gov ↗ ← All trials in the USA