The Natural History of Mucolipidosis Type IV

Stopped early

Conditions studied: Mucolipidosis Type IV

In brief

The purpose of this study is to define the natural history of Mucolipidosis Type IV and identify potential clinical outcome measures.

Key facts

Study ID

NCT01067742

Run by

Baylor Research Institute

People needed

7

Starts

2009-02-19

Expected to finish

2021-07-07

Last updated by the study team

2026-01-26

Who can join

Age: 1 and older, up to 64. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

Where it is running

• Baylor Institute of Metabolic Disease — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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