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Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)

Phase 1
6 Months
3 Years
Open (Enrolling)
Hurler Syndrome

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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:


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:


Principal Investigator

Paul Orchard, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of Minnesota Medical Center


United States: Food and Drug Administration

Study ID:




Start Date:

January 2008

Completion Date:

December 2015

Related Keywords:

  • Hurler Syndrome
  • Hurler Syndrome
  • mucopolysaccharidosis type I
  • Iduronidase deficiency
  • Mucopolysaccharidosis I
  • Mucopolysaccharidoses



University of Minnesota, Fairview Minneapolis, Minnesota  55455