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

February 20, 2013 updated by: Patricia I. Dickson, M.D.

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

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Finland
      • Helsinki, Finland, FI-00014
        • Helsinki University Central Hospital
  • United States
    • California
      • Torrance, California, United States, 90502
        • Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed )

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Hurler-Scheie,Scheie form of MPS I, of Hurler 2 years after hematopoietic stem cell transplantation
  • Spinal cord compression
  • Age greater than 8 years
  • Able to provide legal informed consent
  • Aware of clinical treatment option of observation without treatment or surgical decompression
  • Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only)
  • Currently using two acceptable methods of birth control (non-sterile females of child-bearing potential who are sexually active only)
  • Willing and able to comply with study procedures

Exclusion Criteria:

  • Severe (Hurler) form of MPS I
  • Desires surgical or medical treatment of spinal cord compression
  • Spinal cord compression that warrants immediate surgical intervention
  • Pregnancy or lactation
  • Hematopoietic stem cell transplantation within 2 years of study enrollment
  • Receipt of an investigational drug within 30 days of enrollment
  • Infusion reactions to laronidase that required medical intervention, prophylaxis, or altered enzyme administration
  • Significant anti-iduronidase antibody titer
  • Recent initiation of intravenous laronidase (within past 6 months)
  • Presence of cervical subluxation or similar external pathology as the major cause of cord compression symptoms for which surgical intervention should be immediately undertaken

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: intrathecal laronidase
laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months
Drug: laronidase
0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.
Other Names:
  • Aldurazyme

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month
Time Frame: four months
four months

Secondary Outcome Measures

Outcome Measure
Time Frame
improvement in spinal cord compression due to mucopolysaccharidosis I
Time Frame: four months
four months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Patricia I. Dickson, M.D.

Collaborators

University of California, Los Angeles
FDA Office of Orphan Products Development
The Ryan Foundation

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2005

Primary Completion (Actual)

October 1, 2011

Study Completion (Actual)

October 1, 2011

Study Registration Dates

First Submitted

September 19, 2005

First Submitted That Met QC Criteria

September 19, 2005

First Posted (Estimate)

September 22, 2005

Study Record Updates

Last Update Posted (Estimate)

February 21, 2013

Last Update Submitted That Met QC Criteria

February 20, 2013

Last Verified

February 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MIRC-001
  • 12069-01 (Other Identifier: Los Angeles Biomedical Research Institute at Harbor-UCLA)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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