Patients with Niemann-Pick disease type C (NPC) and GM1 or GM2 gangliosidosis can join the international phase 3 clinical trial NAVIGATE, sponsored by Azafaros. The study evaluates the safety and efficacy of the experimental drug nizubaglustat.
About the NAVIGATE study
The study is designed as a randomised, double-blind, placebo-controlled phase 3 clinical trial. Nizubaglustat is given as an orally dispersible tablet. Participants are assigned in a 2:1 ratio — two thirds receive the treatment and one third placebo. Each patient takes part in the study for 18 months. Around 35 centres in 15 countries are involved — in the USA, Europe and Latin America.
Who can take part
- a diagnosis of the late infantile or juvenile form of Niemann-Pick disease type C, GM1 gangliosidosis or GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease),
- aged at least 4 years,
- the patient must not be taking any treatment that would affect the results of the study.
Support for participants
Costs of travel, accommodation and meals are covered as part of taking part in the study — a great help for families who have to visit specialist centres repeatedly.
Why this matters
Clinical trials are essential for developing treatments for rare diseases — they expand knowledge of how the disease progresses and what therapeutic options exist.