Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring mucopolysaccharidosis, Hurler-Scheie, Scheie, laronidase, spinal cord compression, central nervous system, enzyme replacement therapy, intrathecal, LA Biomed
Eligibility 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
Sites / Locations
- Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed )
- Helsinki University Central Hospital
Arms of the Study
Arm 1
Experimental
intrathecal laronidase
laronidase dose 1.74 mg, route intrathecal, frequency every 30 days, duration three months