Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Status:
Terminated
Trial end date:
2011-10-01
Target enrollment:
Participant gender:
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 to deficiency of the enzyme alpha-l-iduronidase. Spinal cord compression occurs
in this condition due to accumulation of material called glycosaminoglycans (GAG). Laronidase
is the manufactured form of the enzyme alpha-l-iduronidase that is deficient in
mucopolysaccharidosis I patients. The aim of this study is to determine whether laronidase is
safe and effective when given into the spinal fluid as a potential non-surgical treatment for
spinal cord compression due to mucopolysaccharidosis I disease. Funding Source -- FDA OOPD