Expired Study
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Minneapolis, Minnesota 55455


This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.

Study summary:

Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap").


Inclusion Criteria: - Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines. Exclusion Criteria: - Patients are less than 6 months old, or older than 3 years of age. - There is a history of clinically-severe hypersensitivity to Laronidase. - There is a contraindication for repeated lumbar puncture. - The family is not willing to undergo the necessary procedures and evaluations inherent in the study. - Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.



Primary Contact:

Principal Investigator
Paul Orchard, MD
University of Minnesota Medical Center

Backup Contact:


Location Contact:

Minneapolis, Minnesota 55455
United States

There is no listed contact information for this specific location.

Site Status: N/A

Data Source: ClinicalTrials.gov

Date Processed: February 04, 2019

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