Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Completed
Conditions studied: Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
In brief
Hypothesis: Children diagnosed with a lysosomal disease will exhibit developmental, adaptive, and behavioral strengths and difficulties depending upon 1) biomedical risk factors (i.e. the specific genetic disorder responsible for the illness); 2) available modifying interventions, whether medical or behavioral; and 3) social risks in the children's families, neighborhoods and communities. A valid and reliable telephone-based surveillance system can successfully collect the data required to elucidate these developmental, adaptive and behavioral strengths and difficulties.
Key facts
- Study ID
- NCT01938014
- Run by
- University of Chicago
- People needed
- 19
- Starts
- 2009-01-01
- Expected to finish
- 2016-07-23
- Last updated by the study team
- 2019-10-04
Who can join
Age: 0 and older, up to 18. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Children aged 1 to 84 months who have been diagnosed with MPS types I, II, III or VI. Children aged 1 to 84 months who have been diagnosed with some other lysosomal disease. Children aged birth to 18 years who have been diagnosed with Krabbe disease, or who have a positive screening for Krabbe disease.
You may not qualify if…
- Children who do not have a lysosomal disease are excluded from this study.
Where it is running
- University of Chicago — Chicago, Illinois, United States
- University of Minnesota — Minneapolis, Minnesota, United States
- Hunter James Kelly Institute — Buffalo, New York, United States
Full record on ClinicalTrials.gov
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