NAVIGATE: a phase 3 international clinical trial for patients with NPCs and GM1/GM2 gangliosidos
NAVIGATE: a phase 3 international clinical trial for patients with NPCs and GM1/GM2 gangliosidos
Patients with Niemann-Pick disease type C (NPC) and GM1 or GM2 gangliosidos may be enrolled in the international Phase 3 clinical trial NAVIGATE, sponsored by Azafaros. The study evaluates the safety and efficacy of the experimental drug nizubaglustat. This study represents an important step in research into rare neurodegenerative diseases and brings hope to expand future treatment options for patients and their families.
O NAVIGATE studies
The NAVIGATE study is designed as a randomised, double-blind, placebo-controlled, phase 3 clinical trial. Nizubaglustat is administered as an oral dispersible tablet. Patients are enrolled in the study in a 2:1 ratio, which means that two-thirds of the participants receive study treatment and one-third receive placebo. The duration of each patient's participation in the study is 18 months. Approximately 35 centers in 15 countries around the world are involved in the project, including workplaces in the USA, Europe and Latin America.
Who can participate in the study
The study is intended for patients diagnosed with: • late infantile or juvenile forms of Niemann-Pick disease type C (NPC), • GM1 gangliosidos, • GM2 gangliosidos, including Tay-Sachs and Sandhoff disease. The condition for inclusion is the age of 4 years and more. At the same time, due to the strict rules of the study, it is necessary that the participant does not take treatment that could affect the evaluation of the effect of the study drug and thus distort the results of the study.
Organizational support
The study also includes support for families in terms of organization. Costs associated with travel, accommodation and meals are covered as part of participation in the study. This is important for many families, especially with regard to repeated checks and the need to travel to a specialized center.
Participation of our members
We are pleased that three of our members have also been included in this important international study. The study in their case is taking place in Bern, Switzerland. All of them have already successfully passed the initial examination and have been officially included in the study. Currently, the first control visits are taking place at them. We see their involvement as an important step not only for themselves and their families, but also as a valuable contribution to further research into these rare diseases.
Why clinical trials are important
Clinical trials are an essential part of developing new treatment options. In rare diseases such as NPC and GM1/GM2 gangliosidos, they are of particular importance because they help to expand knowledge about the course of the disease and about the possibilities of future therapy. We greatly appreciate all patients and families involved in clinical research. Their participation requires courage, trust and great personal commitment.