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:


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