A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue to receive continued BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.


Study Type: Interventional
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
Enrollment: 23
Study Start Date: February 2015
Estimated Primary Completion Date: December 2021 (Final data collection date for primary outcome measure)

Arms:
- Experimental: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)