Intrathecal Enzyme Replacement for Hurler Syndrome

Completed · Phase 1

Conditions studied: Hurler Syndrome

In brief

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.

Key facts

Study ID

NCT00638547

Run by

Masonic Cancer Center, University of Minnesota

People needed

26

Starts

2008-01-02

Expected to finish

2018-11-18

Last updated by the study team

2018-12-03

Who can join

Age: 1 and older, up to 3. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of Minnesota, Fairview — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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