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Mucopolysaccharidosis I (MPS I) Registry
Recruiting
Trial ID: NCT00144794
Purpose
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
Official Title
Mucopolysaccharidosis I (MPS I) Registry
Stanford Investigator(s)
Eligibility
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.
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305