Intrathecal Enzyme Replacement for Hurler Syndrome
This trial is active, not recruiting.
|Sponsor||Masonic Cancer Center, University of Minnesota|
|Start date||January 2008|
|End date||June 2016|
|Trial size||25 participants|
|Trial identifier||NCT00638547, 0707M11762, MT2007-10|
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), 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.
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
time frame: 1 year
To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
time frame: 1 year
To determine brain changes with magnetic resonance imaging
time frame: 1 and 2 years
To determine neurocognitive changes present in patients with Hurler syndrome
time frame: 6, 12, and 24 months
To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained
time frame: through 1 year
Male or female participants from 6 months up to 3 years old.
Inclusion Criteria: - Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines. Exclusion Criteria: - Patients are less than 6 months old, or older than 3 years of age. - There is a history of clinically-severe hypersensitivity to Laronidase. - There is a contraindication for repeated lumbar puncture. - The family is not willing to undergo the necessary procedures and evaluations inherent in the study. - Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.
|Official title||Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)|
|Principal investigator||Paul Orchard, MD|
|Description||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 (also called a "spinal tap").|
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