Trial | NCT00215527  Report issue

Title

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

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Terminated

  • Intervention/Treatment

    Laronidase ...

  • Study Participants

    4
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.
Study Started
Nov 30
2005
Primary Completion
Oct 31
2011
Study Completion
Oct 31
2011
Last Update
Feb 21
2013
Estimate

Drug laronidase

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

  • Other names: Aldurazyme

intrathecal laronidase Experimental

laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months

Criteria 

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
No Results Posted