Trial Information
Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
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").
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.
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
Outcome Time Frame:
1 year
Safety Issue:
No
Principal Investigator
Paul Orchard, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
University of Minnesota Medical Center
Authority:
United States: Food and Drug Administration
Study ID:
MT2007-10
NCT ID:
NCT00638547
Start Date:
January 2008
Completion Date:
December 2015
Related Keywords:
- Hurler Syndrome
- Hurler Syndrome
- mucopolysaccharidosis type I
- Iduronidase deficiency
- Mucopolysaccharidosis I
- Mucopolysaccharidoses
Name | Location |
|---|
| University of Minnesota, Fairview |
Minneapolis, Minnesota 55455 |
