Intrathecal Enzyme Replacement for Hurler Syndrome

• Status: Completed
• Trial end date: 2018-11-18
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Criteria

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.