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

Stopped early · Phase 1

Conditions studied: Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression

In brief

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

Key facts

Study ID

NCT00215527

Run by

Patricia I. Dickson, M.D.

People needed

4

Starts

2005-11-01

Expected to finish

2011-10-01

Last updated by the study team

2013-02-21

Who can join

Age: 8 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

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

Full record on ClinicalTrials.gov

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