A Phase 3 interventional study of AZ-3102 and Placebo in Niemann-Pick Type C Disease, GM1 Gangliosidosis and GM2 Gangliosidosis, sponsored by Azafaros B.V.. Recruiting at 39 sites in 17 countries. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2026-10-27.
Sponsored by Azafaros B.V. · Phase 3, Interventional, and Treatment
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
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) .
37 studies on the registry are indexed under Niemann-Pick Disease, Type C; 8 are open to participants now.
This study's planned enrollment of 147 is above the median of 21 across 27 interventional studies indexed under Niemann-Pick Disease, Type C.
Browse Niemann-Pick Disease, Type C studies →Azafaros B.V. is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
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Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo). (Recruiting)
Drug: AZ-3102 · Drug: Placebo
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)
Drug: AZ-3102 · Drug: Placebo
Oral dispersible tablets
Also known as: nizubaglustat
A matching placebo will be administered in the same regimen as the intervention
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
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Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Nizubaglustat
Niemann-Pick Disease, Type C →
Azafaros B.V.