Clinical Trial: Mucopolysaccharidosis I (MPS I) Registry
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: Mucopolysaccharidosis I (MPS I) Registry
Brief 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 Summary:
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, +65-6431-2548, [email protected]
- In Europe - +31-35-699-1232, [email protected]
- In Latin America - +617-591-5500, [email protected]
- In North America - +617-591-5500, [email protected]
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome: To evaluate the long-term effectiveness of Aldurazyme [ Time Frame: Approximately 17 Years ]
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Sanofi
Dates:
Date Received: September 2, 2005
Date Started: November 2003
Date Completion: December 2020
Last Updated: May 10, 2017
Last Verified: May 2017