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

This study has been terminated.
(Due to slow enrolment.)
Sponsor:
Collaborators:
Ryan Foundation for MPS Children
University of California, Los Angeles
Information provided by (Responsible Party):
Patricia I. Dickson, M.D., Dickson, Patricia I., M.D.
ClinicalTrials.gov Identifier:
NCT00215527
First received: September 19, 2005
Last updated: February 20, 2013
Last verified: February 2013

September 19, 2005
February 20, 2013
November 2005
October 2011   (final data collection date for primary outcome measure)
safety of intrathecal enzyme treatment by blood and spinal fluid tests each month [ Time Frame: four months ] [ Designated as safety issue: Yes ]
  • safety of intrathecal enzyme treatment by blood and spinal fluid tests each month
  • improvement in neurologic signs related to spinal cord compression, by neurologic examination and Japanese Orthopedic Association Scale each month
  • improvement in neurologic symptoms related to spinal cord compression, by subjective assessments and independence of functioning scale each month
  • improvement in mobility, by six-minute walk test each month
  • improvement in spinal cord compression by MRI imaging and somatosensory evoked potentials at baseline and 4 months
  • improvement in lysosomal storage by spinal fluid glycosaminoglycan levels at each treatment
Complete list of historical versions of study NCT00215527 on ClinicalTrials.gov Archive Site
improvement in spinal cord compression due to mucopolysaccharidosis I [ Time Frame: four months ] [ Designated as safety issue: No ]
  • improvement in spinal fluid pressure, by opening pressure measurements at each intrathecal treatment
  • improvement in hydrocephalus and other brain lesions by MRI at baseline and 4 months
Not Provided
Not Provided
 
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

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

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.

Interventional
Phase 1
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Mucopolysaccharidosis I
  • Lysosomal Storage Diseases
  • Spinal Cord Compression
Drug: laronidase
0.58 mg/ml solution for intravenous injection, dose 1.74 mg intrathecally once per month for four injections.
Other Name: Aldurazyme
Experimental: intrathecal laronidase
laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months
Intervention: Drug: laronidase
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
4
October 2011
October 2011   (final data collection date for primary outcome measure)

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
Both
8 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Finland,   United States
 
NCT00215527
MIRC-001, 12069-01
Yes
Patricia I. Dickson, M.D., Dickson, Patricia I., M.D.
Patricia I. Dickson, M.D.
  • Ryan Foundation for MPS Children
  • University of California, Los Angeles
  • FDA Office of Orphan Products Development
Principal Investigator: Patricia I Dickson, M.D. Los Angeles Biomedical Research Institute at Harbor-UCLA
Dickson, Patricia I., M.D.
February 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP