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Intrathecal Enzyme Replacement for Hurler Syndrome

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Masonic Cancer Center, University of Minnesota
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00638547
First received: March 11, 2008
Last updated: March 4, 2014
Last verified: March 2014
  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: 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)

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 ] [ Designated as safety issue: No ]

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 ] [ 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 ]

Estimated Enrollment: 25
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)
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

Ages Eligible for Study:   6 Months to 3 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00638547

Contacts
Contact: Paul Orchard, MD 612-626-2961 orcha001@umn.edu
Contact: Teresa Kivisto, RN 612-273-2924 tkivist1@fairview.org

Locations
United States, Minnesota
University of Minnesota, Fairview Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Tim Krepski, RN    612-273-2800    tkrepsk1@fairview.org   
Contact: Teresa Kivisto, RN    612-273-2924    tkivist1@fairview.org   
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Paul Orchard, MD University of Minnesota Medical Center
  More Information

No publications provided

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00638547     History of Changes
Other Study ID Numbers: MT2007-10, 0707M11762
Study First Received: March 11, 2008
Last Updated: March 4, 2014
Health Authority: United States: Food and Drug Administration

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

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

ClinicalTrials.gov processed this record on November 27, 2014