Overview
Stem Cell Transplant w/Laronidase for Hurler
Status:
Completed
Completed
Trial end date:
2012-11-01
2012-11-01
Target enrollment:
0
0
Participant gender:
All
All
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 that is associated with decreased complications following transplant.Phase:
Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Criteria
Inclusion Criteria:- 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 Criteria:
- Not being considered for University of Minnesota myeloablative HSCT protocol.
- Previous administration of laronidase enzyme
- Second or subsequent HSCT.