Intrathecal Enzyme Replacement for Hurler Syndrome



Status:Completed
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:Any - 3
Updated:12/5/2018
Start Date:January 2, 2008
End Date:November 18, 2018

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Trial Summary
Trial Overview
Inclusion Criteria

Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)

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.

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.
We found this trial at
1
site
University of Minnesota - Fairview
Minneapolis, Minnesota 55454
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Minneapolis, MN
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