Status:
COMPLETED
Stem Cell Transplant w/Laronidase for Hurler
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Mucopolysaccharidosis I
Hurler Syndrome
Eligibility:
All Genders
Up to 7 years
Phase:
PHASE2
Brief Summary
The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden...
Detailed Description
Subjects will receive laronidase once a week intravenously for 10-12 weeks prior to transplant and for approximately 8 weeks after transplant. Laronidase will be given by intravenous infusion (IV) thr...
Eligibility Criteria
Inclusion
- Patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are candidates for first hematopoietic stem cell transplant (HSCT) according to a University of Minnesota myeloablative HSCT protocol.
Exclusion
- Not being considered for University of Minnesota myeloablative HSCT protocol.
- Previous administration of laronidase enzyme
- Second or subsequent HSCT.
Key Trial Info
Start Date :
March 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2012
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT00176891
Start Date
March 1 2004
End Date
November 1 2012
Last Update
July 11 2019
Active Locations (1)
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1
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455