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