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Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Primary Purpose

Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome

Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
laronidase
Sponsored by
Patricia I. Dickson, M.D.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinal Cord Compression focused on measuring mucopolysaccharidosis, Hurler-Scheie, Scheie, laronidase, spinal cord compression, central nervous system, enzyme replacement therapy, intrathecal

Eligibility Criteria

8 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Hurler-Scheie, Scheie form of MPS I, or Hurler 2 years after hematopoietic stem cell transplantation

    • Spinal cord compression
    • Has received intrathecal laronidase previously with good response and no significant safety concerns
    • 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 (nonsterile females of child-bearing potential who are sexually active only)

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

Sites / Locations

  • Los Angeles Biomedical Research Institute at Harbor-UCLA
  • Helsinki University Central Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

intrathecal laronidase

Arm Description

drug laronidase, dose 1.74 mg, route intrathecal, frequency every 30-90 days, duration 1 year

Outcomes

Primary Outcome Measures

safety of intrathecal enzyme treatment by blood and spinal fluid tests; improvement or stabilization in neurologic signs and symptoms of spinal cord compression

Secondary Outcome Measures

Full Information

First Posted
June 17, 2008
Last Updated
February 19, 2013
Sponsor
Patricia I. Dickson, M.D.
Collaborators
The Ryan Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT00786968
Brief Title
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Official Title
An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
Study Type
Interventional

2. Study Status

Record Verification Date
February 2013
Overall Recruitment Status
Terminated
Why Stopped
Due to slow enrolment.
Study Start Date
January 2008 (undefined)
Primary Completion Date
October 2011 (Actual)
Study Completion Date
October 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Patricia I. Dickson, M.D.
Collaborators
The Ryan Foundation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease
Keywords
mucopolysaccharidosis, Hurler-Scheie, Scheie, laronidase, spinal cord compression, central nervous system, enzyme replacement therapy, intrathecal

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)

8. Arms, Groups, and Interventions

Arm Title
intrathecal laronidase
Arm Type
Experimental
Arm Description
drug laronidase, dose 1.74 mg, route intrathecal, frequency every 30-90 days, duration 1 year
Intervention Type
Drug
Intervention Name(s)
laronidase
Other Intervention Name(s)
Aldurazyme, recombinant human alpha-l-iduronidase
Intervention Description
1.74 mg intrathecally every 1-3 months for 1 year
Primary Outcome Measure Information:
Title
safety of intrathecal enzyme treatment by blood and spinal fluid tests; improvement or stabilization in neurologic signs and symptoms of spinal cord compression
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Hurler-Scheie, Scheie form of MPS I, or Hurler 2 years after hematopoietic stem cell transplantation Spinal cord compression Has received intrathecal laronidase previously with good response and no significant safety concerns 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 (nonsterile females of child-bearing potential who are sexually active only) 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Patricia I Dickson, MD
Organizational Affiliation
Los Angeles Biomedical Research Institute at Harbor-UCLA
Official's Role
Principal Investigator
Facility Information:
Facility Name
Los Angeles Biomedical Research Institute at Harbor-UCLA
City
Torrance
State/Province
California
ZIP/Postal Code
90502
Country
United States
Facility Name
Helsinki University Central Hospital
City
Helsinki
Country
Finland

12. IPD Sharing Statement

Learn more about this trial

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

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