Overview

A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Patients With CLN2 Disease

Status:
Enrolling by invitation
Trial end date:
0000-00-00
Target enrollment:
Participant gender:
Summary
This Phase 2 open-label study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration at 300mg every other week (qow) for a period of 96 weeks, in patients with CLN2. The study is designed to assess disease progression in siblings of children enrolled in the 190-201 study.
Phase:
Phase 2
Details
Lead Sponsor:
BioMarin Pharmaceutical