Clinical Trial: Intrathecal Enzyme Replacement for Hurler Syndrome
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
Brief Summary: 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.
Detailed Summary: 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").
Sponsor: Masonic Cancer Center, University of Minnesota
Current Primary Outcome: To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration [ Time Frame: 1 year ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome:
- 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 evaluate memory/encoding [ Time Frame: 12 and 24 months after hematopoietic stem cell transplantation ]
- 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 ]
Information By: Masonic Cancer Center, University of Minnesota
Dates:
Date Received: March 11, 2008
Date Started: January 2, 2008
Date Completion: June 2017
Last Updated: February 17, 2017
Last Verified: February 2017