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

March 11, 2008
March 4, 2014
January 2008
December 2016   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00638547 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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 evaluate memory/encoding [ Time Frame: 12 and 24 months after hematopoietic stem cell transplantation ] [ 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 ]
Not Provided
Not Provided
 
Intrathecal Enzyme Replacement for Hurler Syndrome
Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)

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").

Interventional
Phase 1
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hurler Syndrome
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
Experimental: Intent-to-Treat
All patients who have received at least one dose of Laronidase.
Intervention: Drug: IRT Laronidase
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
25
December 2016
December 2016   (final data collection date for primary outcome measure)

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.
Both
6 Months to 3 Years
No
Contact: Paul Orchard, MD 612-626-2961 orcha001@umn.edu
Contact: Teresa Kivisto, RN 612-273-2924 tkivist1@fairview.org
United States
 
NCT00638547
MT2007-10, 0707M11762
Yes
Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
Not Provided
Principal Investigator: Paul Orchard, MD University of Minnesota Medical Center
Masonic Cancer Center, University of Minnesota
March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP