Intrathecal Enzyme Replacement for Hurler Syndrome

Masonic Cancer Center, University of Minnesota26 enrolled

Overview

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.

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").

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hurler Syndrome

Interventions

IRT LaronidaseDRUG

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

Eligibility

Sex: ALLMin age: 6 MonthsMax age: 3 Years

Medical Language ↔ Plain English

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.

Locations (1)

University of Minnesota, Fairview

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration

Time frame: 1 year

Secondary Outcomes

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

Data from ClinicalTrials.gov

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