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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
Number of Patients Alive at One Year Post Transplant
Time frame: one year
Number of Patients Requiring Ventilator Support at One Year Post Transplant
Time frame: one year
Donor Engraftment
Time frame: Day 100 post transplant
Patients With Grade III-IV Acute GVHD
Time frame: Day 100 post transplant
Reduction in Glycosaminoglycans (GAG)
Data was not collected on this outcome measure and is not available for reporting.
Time frame: Prior to, During and After ERT
Toxicity (Adverse Events) Associated With Infusions of Laronidase
Data was not collected on this outcome measure and is not available for reporting.
Time frame: 1 year post transplant
Development of Anti-iduronidase Antibodies in Serum
Time frame: 1 Year
Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography
Time frame: Baseline, 12 weeks after laronidase, after transplant
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