Batten Disease Clinical Trial
Official title:
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
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.
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.
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