Stem Cell Transplant w/Laronidase for Hurler
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|ClinicalTrials.gov Identifier: NCT00176891|
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : July 11, 2019
Last Update Posted : July 11, 2019
|Condition or disease||Intervention/treatment||Phase|
|Mucopolysaccharidosis I Hurler Syndrome||Procedure: Stem Cell Transplant Drug: Laronidase ERT||Phase 2|
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 (Clinical Trial)|
|Actual Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)|
|Study Start Date :||March 2004|
|Actual Primary Completion Date :||November 2012|
|Actual Study Completion Date :||November 2012|
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: ERT
Drug: Laronidase ERT
Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
Other Name: Aldurazyme®
- 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) [ Time Frame: Prior to, During and After ERT ]Data was not collected on this outcome measure and is not available for reporting.
- Toxicity (Adverse Events) Associated With Infusions of Laronidase [ Time Frame: 1 year post transplant ]Data was not collected on this outcome measure and is not available for reporting.
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|