NCT00787995

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 353 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.

Conditions

• MPS IV A
• Mucopolysaccharidosis IVA
• Morquio A Syndrome

Timeline

• Start date: 2008-10-01
• Primary completion: 2014-07-01
• Completion: 2014-07-01
• First posted: 2008-11-10
• Last updated: 2014-12-08

Locations

15 sites across 10 countries: United States, Argentina, Brazil, Canada, France, Germany, Italy, Netherlands, Taiwan, United Kingdom