Back to resultsIntrathecal Enzyme Replacement for Hurler Syndrome
Primary Purpose
Hurler Syndrome
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
IRT Laronidase
Sponsored by
About this trial
This is an interventional treatment trial for Hurler Syndrome focused on measuring Hurler Syndrome, mucopolysaccharidosis type I, Iduronidase deficiency
Eligibility 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.
Sites / Locations
- University of Minnesota, Fairview
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Intent-to-Treat
Arm Description
All patients who have received at least one dose of Laronidase.
Outcomes
Primary Outcome Measures
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
Secondary Outcome Measures
To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
To determine brain changes with magnetic resonance imaging
To determine neurocognitive changes present in patients with Hurler syndrome
To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained
Full Information
NCT ID
NCT00638547
First Posted
March 11, 2008
Last Updated
November 30, 2018
Sponsor
Masonic Cancer Center, University of Minnesota
1. Study Identification
Unique Protocol Identification Number
NCT00638547
Brief Title
Intrathecal Enzyme Replacement for Hurler Syndrome
Official Title
Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
Study Type
Interventional
2. Study Status
Record Verification Date
November 2018
Overall Recruitment Status
Completed
Study Start Date
January 2, 2008 (Actual)
Primary Completion Date
February 18, 2017 (Actual)
Study Completion Date
November 18, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Masonic Cancer Center, University of Minnesota
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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 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").
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hurler Syndrome
Keywords
Hurler Syndrome, mucopolysaccharidosis type I, Iduronidase deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Intent-to-Treat
Arm Type
Experimental
Arm Description
All patients who have received at least one dose of Laronidase.
Intervention Type
Drug
Intervention Name(s)
IRT Laronidase
Other Intervention Name(s)
Aldurazyme
Intervention Description
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
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
Time Frame
1 year
Title
To determine brain changes with magnetic resonance imaging
Time Frame
1 and 2 years
Title
To determine neurocognitive changes present in patients with Hurler syndrome
Time Frame
6, 12, and 24 months
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paul Orchard, MD
Organizational Affiliation
University of Minnesota Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Minnesota, Fairview
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Intrathecal Enzyme Replacement for Hurler Syndrome
We'll reach out to this number within 24 hrs