FAQ

The NAVIGATE study (NCT07054515) consists of two 18-month double-blind, randomized, Phase 3 studies to assess an investigational oral medication called nizubaglustat compared to placebo (a tablet with no medicine in it). Running in parallel, one study will test nizubaglustat in patients with Niemann-Pick Type C Disease (NPC) and the other study will involve patients diagnosed with GM1 Gangliosidosis or GM2 Gangliosidosis (GM1/GM2).

Double-blind means that out of every two participants in the study, one receives the study drug nizubaglustat and the other will receive placebo. The NAVIGATE study follows a randomized procedure to choose which patients receive placebo. Neither the participant nor the study physican, study staff, or sponsor will know if you are receiving the study drug or placebo.

Investigational means that the medication (nizubaglustat) has not been approved by any regulatory agencies. However, nizubaglustat has been previously tested in clinical studies involving patients with GM2 gangliosidosis and Niemann-Pick disease type C (NPC). The results demonstrated that it is well tolerated.

Clinical drug development is the multi-phase process where new drugs are tested for safety and to see if they are safe and effective to treat particular diseases. A Phase 3 study is run to confirm the effect and monitor side effects in a larger patient population than previous studies. They are often run in multiple centers across different geographies. It is the final and most critical stage of testing where the data collected from the study is used to support an application to regulatory agencies for marketing approval for the drug.

The NAVIGATE Phase 3 studies will last 18 months.

The NAVIGATE Study will study the safety and efficacy of nizubaglustat in treating two types of lysomal diseases, late-infantile and juvenile forms of NPC and late-infantile and juvenile-onset forms of GM1/GM2. The data collected could be used to support an application to regulatory authorities for marketing approval for nizubaglustat.

The study is being conducted in 32 centers worldwide, including the USA, Latin America, Asia, and in Europe. For the latest update on current study centers, please check the website clinicialtrials.gov and search for the study number NCT07054515.

If you are interested in participating in this study, contact your health/care team and discuss the study, and ask for referral to a study center if needed.

Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).

Nizubaglustat has received Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug Designations (ODD) for GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, as well as Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC from the US Food and Drug Administration (FDA). Additionally, nizubaglustat has been awarded Orphan Medicinal Product Designation (OMPD) for the treatment of GM1 and GM2 gangliosidoses by the European Medicines Agency (EMA) and Innovation Passport for the treatment of GM1 and GM2 gangliosidoses from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).

Eligibility to participate in the study can only be decided by the study physician at the site in discussion with you and your family. 

Overall inclusion criteria are:

• Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease.

• Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. 

The complete enrollment criteria can be found at ClinicalTrials.gov but please contact your physician  if you are interested in participating to see if you are eligible. 

If you are eligible and decide to take part in this study, you will be asked to sign an informed consent form. You will receive a copy of the signed form as well as a patient information sheet for your own records. After you have signed the consent form you will have a screening visit at the study center to receive medical tests to check if you can take part in the study. Your study physician will discuss further details about the study with you.

It is important that you discuss your entire medical history including current medications with your own physician.

Nizubaglustat is an oral capsule drug. Please discuss with your study physician for more information.

You should discuss eligibility, including age at onset, in terms of your eligibility to participate with your study physician.

You can talk to your study physician about any questions or concerns you have about this study.