Trial Information

Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)

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) through a catheter and from there to your child's body's cells and
organs to break down the glycosaminoglycans (GAG) buildup.

Prior to starting ERT, subjects will have a complete physical examination, which includes a
complete assessment of your child's airway and lungs. In addition to standard treatment
evaluations and tests, which are done prior to hematopoietic stem cell transplant (HSCT),
subjects will have the following tests: an additional teaspoon of blood for a baseline test
for serum antibodies against laronidase, before and after the fourth dose of laronidase,
the investigators will collect 2 teaspoons of blood for an alpha-L-iduronidase enzyme level;
to watch for side effects to laronidase and the development of antibodies to laronidase,
approximately 2 teaspoons of blood will be collected every 3 weeks while the subject is
receiving laronidase ERT.