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 |
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
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
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
sites
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