Mucopolysaccharidosis I (MPS I) Registry



Status:Recruiting
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:Any
Updated:3/15/2019
Start Date:November 20, 2003
End Date:December 31, 2020
Contact:For site information, send an email with site number to
Email:Contact-Us@sanofi.com

Use our guide to learn which trials are right for you!

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

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, Vivian.liu@genzyme.com

- In Europe - +31-35-699-1232, europe@mpsiregistry.com

- In Latin America - +617-591-5500, help@mpsiregistry.com

- In North America - +617-591-5500, help@mpsiregistry.com

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.
We found this trial at
43
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New Brunswick, New Jersey 08901
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Springfield, Massachusetts 01199
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