Stem Cell Transplant w/Laronidase for Hurler
|Mucopolysaccharidosis I Hurler Syndrome||Procedure: Stem Cell Transplant Drug: Laronidase ERT||Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)|
- Survival at one year and the proportion of patients in need of ventilator support by one year [ Time Frame: one year ]
- Donor engraftment [ Time Frame: Day 100 ]
- Patients with Grade III-IV acute GVHD [ Time Frame: Day 100 ]
- Reduction in glycosaminoglycans (GAG) [ Time Frame: Prior to, During and After ERT ]
- Toxicity (adverse events) associated with infusions of enzyme [ Time Frame: 1 year ]
- 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 ERT, after transplant ]
|Study Start Date:||March 2004|
|Study Completion Date:||October 2011|
|Primary Completion Date:||October 2011 (Final data collection date for primary outcome measure)|
Experimental: Laronidase ERT Treatment
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Procedure: Stem Cell Transplant
enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Other Name: ERTDrug: Laronidase ERT
Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
Other Name: Aldurazyme®
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00176891
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Paul Orchard, MD||Masonic Cancer Center, University of Minnesota|