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Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

NCT00786968 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2013-02-21

No results posted yet for this study

Summary

This is a one-year extension study of the use of laronidase into the spinal fluid to treat spinal cord compression in mucopolysaccharidosis I. Mucopolysaccharidosis I is a rare genetic condition due to deficiency of the enzyme alpha-l-iduronidase. Spinal cord compression occurs in this condition due to accumulation of material called glycosaminoglycans (GAG). Laronidase is the manufactured form of the enzyme alpha-l-iduronidase that is deficient in mucopolysaccharidosis I patients. The aim of this study is to determine whether laronidase is safe and effective when given into the spinal fluid as a potential non-surgical treatment for spinal cord compression due to mucopolysaccharidosis I disease. Funding Source -- FDA OOPD

Conditions

  • Spinal Cord Compression
  • Mucopolysaccharidosis I
  • Hurler-Scheie Syndrome
  • Scheie Syndrome
  • Lysosomal Storage Disease

Interventions

DRUG

laronidase

1.74 mg intrathecally every 1-3 months for 1 year

Sponsors & Collaborators

  • The Ryan Foundation

    collaborator OTHER

  • Patricia I. Dickson, M.D.

    lead INDIV

Principal Investigators

  • Patricia I Dickson, MD · 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
2008-01-31
Primary Completion
2011-10-31
Completion
2011-10-31

Countries

  • United States
  • Finland

Study Locations

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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 NCT00786968 on ClinicalTrials.gov