Stem Cell Transplant w/Laronidase for Hurler
Mucopolysaccharidosis I, Hurler Syndrome
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring Laronidase ERT, Stem cell transplant, storage disease, inborn errors of metabolism, hurler syndrome, glycosaminoglycans, enzyme replacement
Eligibility 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.
Sites / Locations
- Masonic Cancer Center, University of Minnesota
Arms of the Study
Arm 1
Experimental
Laronidase ERT Treatment
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.