NCT00144794

Mucopolysaccharidosis I (MPS I) Registry

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 1,406 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities. The objectives of the Registry are: * To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase) * To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I * To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Detailed description

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact: * In Asia-Pacific - Vivian Liu, [phone], [e-mail] * In Europe - [phone], [e-mail] * In Latin America - [phone], [e-mail] * In North America - [phone], [e-mail]

Conditions

• Mucopolysaccharidosis I (MPS I)

Timeline

• Start date: 2003-11-20
• Primary completion: 2024-12-31
• Completion: 2024-12-31
• First posted: 2005-09-05
• Last updated: 2026-02-27

Locations

141 sites across 37 countries: United States, Argentina, Australia, Belgium, Brazil, Canada, Chile, Colombia, Czechia, Denmark, Egypt, France, Germany, Hong Kong, India, Indonesia, Ireland, Italy, Japan, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Portugal, Romania, Russia, Saudi Arabia, Slovakia, South Korea, Sweden, Taiwan, Thailand, United Kingdom, Vietnam