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

Not Applicable

Recruiting

• Conditions: Niemann-Pick Type C Disease; GM1 Gangliosidosis; GM2 Gangliosidosis
• Interventions: Drug: AZ-3102; Drug: Placebo

• Registration Number: NCT07054515
• Lead Sponsor: Azafaros A.G.

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 and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543.

Study Timeline

• Study First Submitted: 2025-06-27
• Study First Submitted QC: 2025-06-27
• Study Start: 2025-06-30
• Study First Posted: 2025-07-08
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-04
• Last Update Posted: 2025-08-07
• Primary Completion: 2027-11-04
• Study Completion: 2027-11-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 147

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Subprotocol NCT07082725: Intervention Group NPC	AZ-3102	Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Subprotocol NCT07082725: Intervention Group NPC	Placebo	Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Subprotocol NCT07082543: Intervention Group GM1/GM2	AZ-3102	Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Subprotocol NCT07082543: Intervention Group GM1/GM2	Placebo	Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (32)

Children's Hospital and Research Center at Oakland; 🇺🇸 Oakland, California, United States

Mayo Clinic Childrens Center - PIN; 🇺🇸 Rochester, Minnesota, United States

Lysosomal Rare Disorders Research and Treatment Center; 🇺🇸 Fairfax, Virginia, United States

Hospital Universitario Austral; 🇦🇷 Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina

Hospital de Niños de La Santisima Trinidad; 🇦🇷 Córdoba, Argentina

Women's and Children's Hospital; 🇦🇺 North Adelaide, South Australia, Australia

Royal Melbourne Hospital; 🇦🇺 Parkville, Victoria, Australia

Royal Children's Hospital Melbourne - PIN; 🇦🇺 Parkville, Victoria, Australia

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS; 🇧🇷 Porto Alegre, Rio Grande Do Sul, Brazil

Hospital Pequeno Príncipe; 🇧🇷 Curitiba, Brazil

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