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Clinical Trial Summary

The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.


Clinical Trial Description

The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease. This is an open label Phase 1/2 study conducted in patients with CLN2 disease. Efficacy measures (disease rating scale and MRI) will be compared to a natural history control.

The study will be conducted under cGCP and patients will be closely monitored. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT01907087
Study type Interventional
Source BioMarin Pharmaceutical
Contact
Status Completed
Phase Phase 1/Phase 2
Start date September 2013
Completion date November 2015

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