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March 1, 2026

NAVIGATE: Phase 3 Study of Nizubaglustat for NPC, GM1, and GM2 Gangliosidosis

A new Phase 3 study, titled NAVIGATE, is currently evaluating the efficacy and safety of Nizubaglustat for patients with late-infantile and juvenile forms of Niemann-Pick Type C (NPC) and GM1/GM2 Gangliosidosis.

This global multi-center study includes active sites in India, providing a critical opportunity for local patients to access investigational therapy.

Mechanism of Action

Nizubaglustat is an oral, brain-penetrant small molecule. It acts as a selective inhibitor of:

Glucosylceramide synthase (GCS)
Non-lysosomal neutral glucosylceramidase (NLGase)

By inhibiting these enzymes, the drug aims to reduce the accumulation of toxic substrates characteristic of these neurodegenerative lysosomal storage diseases.

Study Design

The NAVIGATE trial utilizes two subprotocols conducted identically but evaluated independently:

NPC Cohort: Aiming for 72 participants (not currently using miglustat).
GMx (GM1/GM2) Cohort: Aiming for 75 participants.

The study features an 18-month double-blind main period with a 2:1 randomization (Nizubaglustat to Placebo).

Following the initial 18 months, participants in the placebo arm transition to receiving Nizubaglustat.

Key Inclusion Criteria

Age/Weight: 4 years or older and weighing at least 10 kg.
Diagnosis: Genetically or enzymatically confirmed NPC, GM1, or GM2.
Disease Onset: NPC: Neurological onset between 2–15 years of age. GMx: Neurological onset between 1–10 years of age.
Clinical Score: A total Scale for the Assessment and Rating of Ataxia (SARA) score between 3 and 30 points.
Medication Constraints: Miglustat is not allowed during the study. Other approved non-investigational drugs are permitted if doses are stable before baseline.