A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
Summary
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2. The study is designed to assess disease progression in CLN2 patients treated with BMN 190 compared to natural history data from untreated historical controls.
Timeline
- Start
- 2016-01-22
- Primary completion
- 2022-04-20
- Completion
- 2022-04-20
Outcome
Met primary endpoint
registry analysis (superiority test); BMN 190-203 vs Natural History Comparator: 190-901 Participants; p < 0.0001; t-test, unequal variance NCT02678689 ↗
registry analysis (superiority test); BMN 190-203 vs Natural History Comparator: 190-901 Participants; p <.0001; Hazard Ratio (HR) 0.091 (95% CI 0.021 to 0.393); Cox Model Wald Test NCT02678689 ↗
registry analysis (superiority test); BMN 190-203 vs Natural History Comparator: 190-901 Participants; p = 0.0032; Hazard Ratio (HR) 0.000 (95% CI 0.000 to 0.000); Cox Model Wald Test NCT02678689 ↗
registry analysis (superiority test); BMN 190-203 vs Natural History Comparator: 190-901 Participants; p <.0001; t-test, unequal variance NCT02678689 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cerliponase Alfa | Protein / enzyme biologic | — | Other |