Status:
TERMINATED
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Lead Sponsor:
Patricia I. Dickson, M.D.
Collaborating Sponsors:
The Ryan Foundation
Conditions:
Spinal Cord Compression
Mucopolysaccharidosis I
Eligibility:
All Genders
8+ years
Phase:
PHASE1
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 t...
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 centr...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2011
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00786968
Start Date
January 1 2008
End Date
October 1 2011
Last Update
February 21 2013
Active Locations (2)
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1
Los Angeles Biomedical Research Institute at Harbor-UCLA
Torrance, California, United States, 90502
2
Helsinki University Central Hospital
Helsinki, Finland