Mucopolysaccharidosis I (MPS I) Registry
Mucopolysaccharidosis I (MPS I)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
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Participation Requirements
-
Sex:
ALL -
Eligible Ages:
0 and up
Participation Criteria
Inclusion Criteria:
* All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha (a)-L-iduronidase enzyme activity and/or B. mutation(s) in the gene coding for a-L-iduronidase, or measurable clinical signs and symptoms of MPS I
* For all patients there should be a completed patient authorization form
Exclusion Criteria:
* No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.
Study Location
Investigational Site Number : 124014
Investigational Site Number : 124014Calgary, Alberta
Canada
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Investigational Site Number : 124022
Investigational Site Number : 124022Moncton, New Brunswick
Canada
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Investigational Site Number : 124001
Investigational Site Number : 124001Sherbrooke, Quebec
Canada
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Investigational Site Number : 124019
Investigational Site Number : 124019Winnipeg, Manitoba
Canada
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Investigational Site Number : 124018
Investigational Site Number : 124018Montreal, Quebec
Canada
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Investigational Site Number : 124002
Investigational Site Number : 124002Edmonton, Alberta
Canada
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Investigational Site Number : 124021
Investigational Site Number : 124021London, Ontario
Canada
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Investigational Site Number : 124011
Investigational Site Number : 124011Vancouver, British Columbia
Canada
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Investigational Site Number : 124006
Investigational Site Number : 124006Toronto, Ontario
Canada
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- Study Sponsored By
- Sanofi
- Participants Required
- More Information
- Study ID:
NCT00144794