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

NCT00215527 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2013-02-21

No results posted yet for this study

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

Conditions

  • Mucopolysaccharidosis I
  • Lysosomal Storage Diseases
  • Spinal Cord Compression

Interventions

DRUG

laronidase

0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.

Sponsors & Collaborators

  • The Ryan Foundation

    collaborator OTHER

  • University of California, Los Angeles

    collaborator OTHER

  • FDA Office of Orphan Products Development

    collaborator FED

  • Patricia I. Dickson, M.D.

    lead INDIV

Principal Investigators

  • Patricia I Dickson, M.D. · Los Angeles Biomedical Research Institute at Harbor-UCLA

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-11-30
Primary Completion
2011-10-31
Completion
2011-10-31

Countries

  • United States
  • Finland

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00215527 on ClinicalTrials.gov