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

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

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07054515

Enrollment

147

Registered

2025-07-08

Start date

2025-06-30

Completion date

2028-06-14

Last updated

2026-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

GM1 Gangliosidosis, GM2 Gangliosidosis, Niemann-Pick Type C Disease

Keywords

Nizubaglustat

Brief summary

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

Detailed description

This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis. Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols. For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 (Recruiting) and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543 (Active, not recruiting - No longer looking for participants) .

Interventions

DRUGAZ-3102

Oral dispersible tablets

DRUGPlacebo

A matching placebo will be administered in the same regimen as the intervention

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Masking description

Participants, investigators, other study personnel at the site, and the Sponsor will be blinded

Eligibility

Sex/Gender

ALL

Age

4 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
Number of participants allocated to subprotocol AZA-001-301-NPC	Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx	Baseline to month 18

Countries

Argentina, Australia, Brazil, Canada, France, Germany, India, Italy, Mexico, Pakistan, Portugal, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom, United States

Contacts

CONTACTPatient Advocacy Representative

info@azafaros.comPlease reach out by email

CONTACTContact for Healthcare Professionals

medinfo@azafaros.comPlease reach out by email

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 17, 2026