Status:
COMPLETED
Intrathecal Enzyme Replacement for Hurler Syndrome
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Hurler Syndrome
Eligibility:
All Genders
6-3 years
Phase:
PHASE1
Brief 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, i...
Detailed Description
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 ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
January 2 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 18 2018
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT00638547
Start Date
January 2 2008
End Date
November 18 2018
Last Update
December 3 2018
Active Locations (1)
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1
University of Minnesota, Fairview
Minneapolis, Minnesota, United States, 55455