NCT00215527

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Patricia I. Dickson, M.D. · Individual
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

Summary

The investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of mucopolysaccharidosis I (MPS I). Funding source -- FDA OOPD

Detailed description

Enzyme replacement therapy (ERT) has been developed for mucopolysaccharidosis I (MPS I), a lysosomal storage disorder. ERT helps many physical ailments due to the disease, but does not treat the central nervous system, due to inability to cross the blood brain barrier. Our purpose is to test delivery of ERT to the spinal fluid via intrathecal injection in patients with MPS I. In this pilot study, we will use recombinant human α-L-iduronidase administered intrathecally once per month for four months to individuals with the Hurler-Scheie and Scheie forms of MPS I and spinal cord compression. If successful, intrathecal delivery could represent a practical, straightforward method of treating central nervous system disease due to lysosomal storage.

Conditions

• Mucopolysaccharidosis I
• Lysosomal Storage Diseases
• Spinal Cord Compression

Interventions

Type	Name	Description
DRUG	laronidase	0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.

Timeline

• Start date: 2005-11-01
• Primary completion: 2011-10-01
• Completion: 2011-10-01
• First posted: 2005-09-22
• Last updated: 2013-02-21

Locations

2 sites across 2 countries: United States, Finland