26
Participants
Start Date
January 2, 2008
Primary Completion Date
February 18, 2017
Study Completion Date
November 18, 2018
IRT Laronidase
"Laronidase belongs to a class of drugs called enzyme replacement therapies or ERT that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingredient in laronidase is a protein that is identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup.~Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture"
University of Minnesota, Fairview, Minneapolis
Masonic Cancer Center, University of Minnesota
OTHER