NCT00852358
MPS 1, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Cognitive Decline, Scheie Syndrome, Lysosomal Storage Disease
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
This is a 24-month study of the use of laronidase administered into the spinal fluid to treat cognitive decline in mucopolysaccharidosis I (MPS I). MPS I is a rare genetic condition due to deficiency of the enzyme alpha-l-iduronidase. Laronidase is the manufactured form of the enzyme alpha-l-iduronidase.
MPS I is a heterogeneous disease with several clinical phenotypes ranging from the most severe, Hurler syndrome, to the attenuated forms, Hurler-Scheie and Scheie. Although patients with milder forms of MPS I may not have grossly observable problems with cognition, these patients do have learning difficulties that are apparent in school and with neuropsychological testing. The goal of this study is to evaluate whether intrathecal recombinant human alpha-l-iduronidase (rhIDU) injections can stabilize or improve cognitive decline in individuals with MPS I.
All
From 6 Years
No
laronidase
Na
Interventional
9
2009-06
2016-01-26
Oakland, California, United States
Torrance, California, United States
Minneapolis, Minnesota, United States
1. A score of one standard deviation below mean on IQ testing or in one domain of neuropsychological function (language, memory, or non-verbal ability), OR
2. Documented historical evidence of a decline of greater than one standard deviation on sequential testing, OR
3. A score between 0.75 and 1 standard deviation below the mean, AND the cognitive deficit affects daily performance.
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