©2025 Stanford Medicine
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