A Phase 2/3 interventional study of AZ-3102 in Niemann-Pick Disease Type C, GM2 Gangliosidosis and GM1 Gangliosidosis, sponsored by Azafaros B.V.. Not yet recruiting. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Azafaros B.V. · Phase 2/3, Interventional, and Treatment
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to continue nizubaglustat treatment.
This is a multi-center, global, open-label, extension trial in participants with Niemann-Pick disease type C (NPC), GM1 gangliosidosis, GM2 gangliosidosis, or Gaucher disease type 3 (GD3) who previously participated in a clinical trial of nizubaglustat (AZ-3102) and wish to continue treatment. All participants will receive nizubaglustat once daily.
The primary objective is to assess the long-term safety and tolerability of continued daily administration of nizubaglustat. The trial will also investigate the effect of nizubaglustat on disease biomarkers and explore the effect of long-term treatment on neurological aspects of disease progression.
Participants may remain in the trial under marketing authorization has been granted for nizubaglustat in their country or until the sponsor decides to stop the trial.
54 studies on the registry are indexed under Niemann-Pick Disease, Type C; 9 are open to participants now.
This study's planned enrollment of 218 is above the median of 25 across 38 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.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Drug: AZ-3102
Daily oral intake of nizubaglustat (AZ-3102) dispersible tablets
The Incidence and severity of treatment-emergent adverse events (TEAEs)
Change in vital signs parameters over time
Time frame: Through study completion, an average of 4 years
The Incidence and severity of treatment-emergent adverse events (TEAEs)
Change in laboratory safety parameters over time.
Time frame: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Plasma lyso-monosialoganglioside GM1 (Lyso-GM1; for participants with GM1) since the first dose of nizubaglustat
Time frame: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Plasma Lyso-GM2 (for participants with GM2) since the first dose of nizubaglustat
Time frame: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Plasma N-palmitoyl-O-phosphocholine-serine (PPCS; for participants with NPC) since the first dose of nizubaglustat
Time frame: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Glucosylsphingosine (GlcSph; for participants with GD3) since the first dose of nizubaglustat
Time frame: Through study completion, an average of 4 years
No study locations are listed for this record.
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 ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Nizubaglustat
Niemann-Pick Disease, Type C →
Azafaros B.V.