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Oral nizubaglustat (AZ-3102) for late‑infantile and juvenile Niemann‑Pick C, GM1, and GM2 gangliosidoses

18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Phase 3 Interventional Azafaros A.G. · NCT07054515

This 18‑month study will test whether daily oral nizubaglustat (AZ‑3102) helps people age 4 and up with late‑infantile or juvenile Niemann‑Pick C, GM1, or GM2 gangliosidosis compared with placebo.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment147 (estimated)
Ages4 Years and up
SexAll
SponsorAzafaros A.G. Industry-sponsored
Locations37 sites (Oakland, California and 36 other locations)
Trial IDNCT07054515 on ClinicalTrials.gov

What this trial studies

This randomized, double‑blind, placebo‑controlled Phase 3 Master Protocol tests oral nizubaglustat (AZ‑3102) over 18 months in participants with late‑infantile or juvenile NPC, GM1, or GM2 gangliosidosis. Participants are assigned to disease‑specific subprotocols and randomized to receive active drug or placebo, with detailed procedures and endpoints defined per subprotocol. The study is multicenter and includes planned safety monitoring, efficacy assessments, and disease‑specific outcome measures. Specific eligibility and exclusion criteria differ by subprotocol and are described in the corresponding clinicaltrials.gov records.

Who should consider this trial

Good fit: Children and adolescents age 4 years and older with a confirmed diagnosis of late‑infantile or juvenile‑onset Niemann‑Pick C, GM1, or GM2 gangliosidosis who meet the disease‑specific subprotocol criteria.

Not a fit: Patients with adult‑onset disease, very advanced irreversible neurological damage, or who do not meet subprotocol eligibility (including specific medical exclusions) are unlikely to receive benefit from this intervention.

Why it matters

Potential benefit: If successful, nizubaglustat could slow neurodegeneration and help preserve neurological function and quality of life in affected children and adolescents.

How similar studies have performed: Related substrate‑reduction therapies such as miglustat have shown mixed or limited benefits in NPC and are less established for GM1/GM2, so the approach has some precedent but remains incompletely proven.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
* 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. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).

Exclusion Criteria:

* Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
* Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Where this trial is running

Oakland, California and 36 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 GangliosidosisNizubaglustat
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.