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Sponsors and Collaborators: |
University of Minnesota Masonic Cancer Center, University of Minnesota |
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Information provided by: | University of Minnesota |
ClinicalTrials.gov Identifier: | NCT00638547 |
This protocol will examine whether the enzyme -L-iduronidase, delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.
Condition | Intervention | Phase |
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MPS IH (Hurler Syndrome) |
Drug: IRT Laronidase |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome) |
Estimated Enrollment: | 25 |
Study Start Date: | December 2007 |
Estimated Study Completion Date: | December 2014 |
Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
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
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 (performed on another study protocol). This procedure is done by lumbar puncture (also called a "spinal tap").
Ages Eligible for Study: | 8 Months to 3 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Paul Orchard, MD | 612-626-1926 | orcha001@umn.edu |
Contact: Teresa Kvisto, RN | 612-273-2800 | tkvist1@fairview.org |
United States, Minnesota | |
University of MInnesota, Fairview | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Tim Krepski, RN 612-273-2800 | |
Contact: Teresa Kvisto, RN 612-273-2800 tkvist!@fairview.org |
Principal Investigator: | Paul Orchard, MD | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota ( Paul Orchard, M.D. ) |
Study ID Numbers: | 0707M11762, MT2007-10 |
Study First Received: | March 11, 2008 |
Last Updated: | September 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00638547 |
Health Authority: | United States: Food and Drug Administration |
Alpha-L-iduronidase deficiency Metabolism, Inborn Errors Mucopolysaccharidoses Metabolic Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases |
Connective Tissue Diseases Mucopolysaccharidosis Metabolic disorder Hurler syndrome Mucopolysaccharidosis I |
Pathologic Processes Disease Syndrome Mucinoses Carbohydrate Metabolism, Inborn Errors |