Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders

NCT07885917 — Phase 2 / Phase 3, Not yet recruiting, 218 participants, Azafaros B.V.

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An Open-label, Roll-over Trial to Evaluate the Long-term Safety and Tolerability of Nizubaglustat (AZ-3102) in Participants With Rare Lysosomal Disorders Who Have Participated in Previous Nizubaglustat Trials and Wish to Continue Nizubaglustat Treatment

Registry	NCT07885917, acronym HARBOR
Status	Not yet recruiting
Phase	Phase 2 / Phase 3
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	218 (Estimated)
Sponsor	Azafaros B.V. (Industry)
Conditions	Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
Interventions	AZ-3102 (Drug)
Dates	start 2026-12-10 (estimated), primary completion 2030-04-01 (estimated), completion 2030-04-01 (estimated), first posted 2026-10-21, last updated 2026-10-21

-Summary

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.

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+Detailed description

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.

+Arms and interventions

Arm	Type	Interventions	Description
All participants will receive nizubaglustat	Experimental	Drug: AZ-3102

AZ-3102 (Drug): Daily oral intake of nizubaglustat (AZ-3102) dispersible tablets

+Outcome measures

Primary outcomes

The Incidence and severity of treatment-emergent adverse events (TEAEs) [Through study completion, an average of 4 years]: Change in vital signs parameters over time
The Incidence and severity of treatment-emergent adverse events (TEAEs) [Through study completion, an average of 4 years]: Change in laboratory safety parameters over time.

Secondary outcomes

Change in concentrations [Through study completion, an average of 4 years]: Absolute and percent change in the concentrations of Plasma lyso-monosialoganglioside GM1 (Lyso-GM1; for participants with GM1) since the first dose of nizubaglustat
Change in concentrations [Through study completion, an average of 4 years]: Absolute and percent change in the concentrations of Plasma Lyso-GM2 (for participants with GM2) since the first dose of nizubaglustat
Change in concentrations [Through study completion, an average of 4 years]: 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
Change in concentrations [Through study completion, an average of 4 years]: Absolute and percent change in the concentrations of Glucosylsphingosine (GlcSph; for participants with GD3) since the first dose of nizubaglustat

+Eligibility

Sex: All, ages: 4 Years to any

Inclusion Criteria:

* Have participated in a trial of nizubaglustat and wish to continue nizubaglustat treatment
* Participation is considered beneficial by the investigator
* Female participants of childbearing potential who are willing to follow the protocol-defined contraceptive requirements
* Male participants who are willing to follow the protocol-defined contraceptive requirements

Exclusion Criteria:

* A positive serum pregnancy test (for women of childbearing potential).
* Any medical event or condition that prevents participation in the trial based on the judgment of the investigator (e.g., severe infection and severe cognitive impairment).
* Use of any other treatment with a similar mode of action to nizubaglustat (GCS or NLGase inhibition), e.g., miglustat or venglustat.
* Participation in another interventional trial or non-interventional study or in an Early Access Program.