The Natural History of Mucolipidosis Type IV (NCT01067742) | Clinical Trial Compass
TerminatedNot Applicable
The Natural History of Mucolipidosis Type IV
Stopped: PI left site
United States7 participantsStarted 2009-02-19
Plain-language summary
The purpose of this study is to define the natural history of Mucolipidosis Type IV and identify potential clinical outcome measures.
Who can participate
Age range1 Year – 64 Years
SexALL
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Inclusion Criteria:
Subjects must:
* Have a definitive diagnosis of MLIV based at least on a compatible history and significantly elevated blood gastrin levels
* Be able to travel to the Baylor Institute of Metabolic Disease in Dallas and spend 2-3 working days on site
* Be able to tolerate a general exam and neurological exam
* Be able to tolerate a modest amount of blood drawing, provide a urine specimen, and have a skin biopsy(if not previously done)
* Be able to tolerate the performance of necessary neuroimaging studies to include EEG and Head MRI
* Be able to tolerate a neuropsychological testing and rehabilitation evaluation
Exclusion Criteria:
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