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Intrathecal Enzyme Replacement for Hurler Syndrome

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00638547
First Posted: March 19, 2008
Last Update Posted: October 5, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
  Purpose
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.

Condition Intervention Phase
Hurler Syndrome Drug: IRT Laronidase Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)

Resource links provided by NLM:


Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • 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 ]

Enrollment: 26
Actual Study Start Date: January 2, 2008
Estimated Study Completion Date: November 10, 2017
Primary Completion Date: November 10, 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intent-to-Treat
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

Detailed 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").
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 3 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00638547


Locations
United States, Minnesota
University of Minnesota, Fairview
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Paul Orchard, MD University of Minnesota Medical Center
  More Information

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00638547     History of Changes
Other Study ID Numbers: MT2007-10
0707M11762 ( Other Identifier: IRB, University of Minnesota )
First Submitted: March 11, 2008
First Posted: March 19, 2008
Last Update Posted: October 5, 2017
Last Verified: September 2017

Keywords provided by Masonic Cancer Center, University of Minnesota:
Hurler Syndrome
mucopolysaccharidosis type I
Iduronidase deficiency

Additional relevant MeSH terms:
Syndrome
Mucopolysaccharidoses
Mucopolysaccharidosis I
Disease
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases