NCT01515956

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks.

Conditions

• Mucopolysaccharidosis IVA
• Morquio A Syndrome
• MPS IVA

Interventions

Type	Name	Description
DRUG	BMN 110	Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 208 weeks.

Timeline

• Start date: 2011-10-01
• Primary completion: 2016-02-01
• Completion: 2016-02-01
• First posted: 2012-01-24
• Last updated: 2017-08-10
• Results posted: 2017-07-06

Locations

5 sites across 4 countries: United States, Italy, Taiwan, United Kingdom