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A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

RecruitingPhase 3

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

Who can join

Ages 4 and older · All sexes

Full eligibility 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

About the study

An 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

What is being tested

AZ-3102 (drug)
Placebo (drug)

Sponsor: Azafaros B.V. · Participants: 147 · Started: Jun 30, 2025

Contact the study team

Patient Advocacy Representative · Phone: Please reach out by email
Contact for Healthcare Professionals · Phone: Please reach out by email

Official record on ClinicalTrials.gov — NCT07054515

Locations in the U.S.

California	UCSF Children's Hospital and Research Center at Oakland, Oakland
Minnesota	University of Minnesota Medical School, Minneapolis Mayo Clinic Childrens Center - PIN, Rochester
Texas	Children's Medical Center Dallas, Dallas
Virginia	Lysosomal Rare Disorders Research and Treatment Center, Fairfax

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.