Intrathecal Enzyme Replacement for Hurler Syndrome
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)|
- To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
- To determine brain changes with magnetic resonance imaging [ Time Frame: 1 and 2 years ] [ Designated as safety issue: No ]
- To determine neurocognitive changes present in patients with Hurler syndrome [ Time Frame: 6, 12, and 24 months ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: Yes ]
|Study Start Date:||January 2008|
|Estimated Study Completion Date:||December 2016|
|Estimated Primary Completion Date:||December 2016 (Final data collection date for primary outcome measure)|
All patients who have received at least one dose of Laronidase.
Drug: IRT Laronidase
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
Other Name: Aldurazyme
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").
Please refer to this study by its ClinicalTrials.gov identifier: NCT00638547
|Contact: Paul Orchard, MDemail@example.com|
|Contact: Teresa Kivisto, RNfirstname.lastname@example.org|
|United States, Minnesota|
|University of Minnesota, Fairview||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Tim Krepski, RN 612-273-2800 email@example.com|
|Contact: Teresa Kivisto, RN 612-273-2924 firstname.lastname@example.org|
|Principal Investigator:||Paul Orchard, MD||University of Minnesota Medical Center|