Overview

Stem Cell Transplant w/Laronidase for Hurler

Status:
Completed
Trial end date:
2012-11-01
Target enrollment:
0
Participant gender:
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 2
Accepts Healthy Volunteers?
No
Details
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.