Trial ID or NCT#

NCT00144794

Status

RECRUITING

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

Eligibility Criteria

Sexes Eligible for Study: All
Accepts Healthy Volunteers: No

Investigator(s)

Gregory Enns
Geneticist
Professor of Pediatrics (Genetics) at the Lucile Salter Packard Children's Hospital

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CONTACT

SPECTRUM