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
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Enrollment
- 147
- Locations
- 53
- Primary Endpoint
- Number of participants allocated to subprotocol AZA-001-301-NPC
Study Overview
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) .
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Participants, investigators, other study personnel at the site, and the Sponsor will be blinded
Eligibility Criteria
- Ages
- 4 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Subprotocol NCT07082543: Intervention Group GM1/GM2
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
(Active, not recruiting - No longer looking for participants)
Intervention: AZ-3102 (Drug)
Subprotocol NCT07082725: Intervention Group NPC
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
(Recruiting)
Intervention: Placebo (Drug)
Subprotocol NCT07082725: Intervention Group NPC
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
(Recruiting)
Intervention: AZ-3102 (Drug)
Subprotocol NCT07082543: Intervention Group GM1/GM2
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
(Active, not recruiting - No longer looking for participants)
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Number of participants allocated to subprotocol AZA-001-301-NPC
Time Frame: Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx
Time Frame: Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-NPC
Time Frame: Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx
Time Frame: Baseline to month 18
Secondary Outcomes
No secondary outcomes reported