Intrathecal Enzyme Replacement for Hurler Syndrome

University of Minnesota (UMN)

Status and phase

Completed

Phase 1

Conditions

Hurler Syndrome

Treatments

Drug: IRT Laronidase

Study type

Interventional

Funder types

Other

Identifiers

NCT00638547

0707M11762 (Other Identifier)

MT2007-10

Details and patient eligibility

About

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.

Full 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").

Enrollment

26 patients

Sex

All

Ages

6 months to 3 years old

Volunteers

No Healthy Volunteers

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.