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Clinical Trial Summary

The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT01047306
Study type Observational
Source Takeda
Contact
Status Completed
Phase
Start date February 15, 2010
Completion date July 10, 2013

See also
  Status Clinical Trial Phase
Completed NCT01873911 - Neurobehavioral Phenotypes in MPS III