Azafaros Completes Enrollment of Phase 3 (NAVIGATE) Study Evaluating Nizubaglustat for the Treatment of Patients with GM1/GM2 Gangliosidoses

GM2 gangliosidosis

https://www.azafaros.com/news-media/azafaros-completes-enrollment-of-phase-3-navigate-study-evaluating-nizubaglustat-for-the-treatment-of-patients-with-gm1-gm2-gangliosidoses

Azafaros’s Phase 3 NAVIGATE study for treatment of patients with GM1/GM2 Gangliosidosis and Niemann-Pick type C diseasehas completed enrolment of patients with GM1/GM2 Gangliosidosis. The Phase 3 GM1/GM2 study enrolled a minimum of 75 patients across 25 clinical sites in 13 countries. The study is evaluating oral administration of nizubaglustat compared to treatment with placebo . A placebo is a treatment with no active medicine that is often used in research to compare against real treatments

 The trial is over an 18-month treatment period. Efficacy assessments will focus on neurological manifestations and disease progression. All patients treated in the study following the 18-month treatment period are eligible to continue nizubaglustat in an open label extension study, this is when after the study ends, participants are invited to join an open-label extension study. Everyone receives the new drug, this allows researchers continue to monitor safety and effectiveness over a longer period.