This study is testing an oral medication called nizubaglustat (AZ-3102) for people with late-infantile or juvenile forms of Niemann-Pick Type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. Researchers want to see if nizubaglustat is safe and effective. You might receive nizubaglustat or a placebo (an inactive substance that looks like the study drug). The study aims to enroll 147 participants aged 4 years and older. Success will be measured by how many participants are assigned to each disease-specific part of the study over 18 months. The recruitment status for Niemann-Pick Type C is "Recruiting," but for GM1 or GM2 gangliosidosis, it is "Active, not recruiting," meaning they are no longer looking for participants for that group.

Study design

This is a Phase 3, randomized, double-blind (meaning neither you nor your doctor will know if you're receiving the study drug or placebo), placebo-controlled study involving up to 147 participants.

What's involved

Not specified in the trial record.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for 18 months to assess outcomes.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.