A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Condition(s) studied
Investigational drug(s) / intervention(s)
BMN 111: BMN 111 will be administered subcutaneously daily.
Study summary
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.
Eligibility
Primary outcome measure(s)
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] — 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
* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events
Trial sites (9)
| Facility | City | Region | Status |
|---|---|---|---|
| Children's Hospital & Research Center Oakland | Oakland | California | |
| Harbor - UCLA Medical Center | Torrance | California | |
| Ann and Robert H. Lurie Childrens Hospital of Chicago | Chicago | Illinois | |
| Johns Hopkins McKusick - Institute of Genetic Medicine | Baltimore | Maryland | |
| Vanderbilt University | Nashville | Tennessee | |
| Baylor College of Medicine | Houston | Texas | |
| Murdoch Children's Research Institute | Parkville | Victoria | |
| Institut Necker | Paris | France | |
| Guys & St. Thomas NHS Foundation Trust Evelina Hospital | London | United Kingdom |
More BioMarin Pharmaceutical trials in the UK
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 NCT02724228 on ClinicalTrials.gov ↗ ← All trials in the UK