Niemann-Pick type C disease  (NPC) study

What is a clinical research study?

Researchers conduct clinical studies, also known as clinical trials, to learn if an investigational medication or device is safe and effective for people who have certain health conditions. Clinical studies follow strict scientific standards and regulations to ensure they produce the best data for health care decision-making while protecting the participants.

About this study

NAVIGATE is a double-blind, placebo-controlled Phase 3 study to assess an investigational medication called nizubaglustat (an oral dispersible tablet) compared to placebo (a tablet with no medicine in it) for the treatment of NPC.

Double-blind means that neither you nor the study doctor, study staff, or sponsor will know if you are receiving the study drug or placebo. One out of every three patients will receive the placebo.

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

Timelines

The study is divided into the following periods:

• Screening Period (up to 30 days)
  Where you will undergo certain examinations and procedures to see if you can join the study.
• Treatment Period (about 18 months)
  You will receive either the study drug or placebo for 18 months. Each dose will consist of a daily oral dispersible tablet (no need for a central line).
• Open Label Treatment Period
  After attending the Month 18 visit, you will be offered the chance to enter an open-label treatment period until the end of the study. Participants receiving placebo who wish to continue will also switch to nizubaglustat. In the open-label extension, you will have study visits at month 21, 24, and every 6 months thereafter.

• Follow-up Period
  You will receive a follow-up safety phone call from the study site two weeks after the last dose of treatment, to monitor adverse events and to check on your general well-being.

Participants will receive a full reimbursement of all study-related costs.