NCT07885917

Brief 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.

Trial Health

65
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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
218

participants targeted

Target at P75+ for phase_2

Timeline
40mo left

Started Dec 2026

Typical duration for phase_2

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 14, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

October 21, 2026

Completed
2 months until next milestone

Study Start

First participant enrolled

December 10, 2026

Expected
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2030

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2030

Last Updated

October 21, 2026

Status Verified

October 1, 2026

Enrollment Period

3.3 years

First QC Date

October 14, 2026

Last Update Submit

October 14, 2026

Conditions

Niemann-Pick Disease Type CGM2 GangliosidosisGM1 GangliosidosisGaucher Disease Type 3

Keywords

NPCGM1GM2GD3NizubaglustatHARBORRare Lysosomal DisordersAZ-3102GM1 gangliosidosisGM2 gangliosidosisGaucher Disease Type 3Niemann-Pick disease type C

Outcome Measures

Primary Outcomes (2)

  • The Incidence and severity of treatment-emergent adverse events (TEAEs)

    Change in vital signs parameters over time

    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.

    Through study completion, an average of 4 years

Secondary Outcomes (4)

  • Change in concentrations

    Through study completion, an average of 4 years

  • Change in concentrations

    Through study completion, an average of 4 years

  • Change in concentrations

    Through study completion, an average of 4 years

  • Change in concentrations

    Through study completion, an average of 4 years

Study Arms (1)

All participants will receive nizubaglustat

EXPERIMENTAL
Drug: AZ-3102

Interventions

AZ-3102DRUG

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

All participants will receive nizubaglustat

Eligibility Criteria

Age4 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Niemann-Pick Disease, Type CGangliosidoses, GM2Gangliosidosis, GM1Gaucher Disease

Condition Hierarchy (Ancestors)

Niemann-Pick DiseasesSphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHistiocytosis, Non-Langerhans-CellHistiocytosisLymphatic DiseasesHemic and Lymphatic DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism DisordersGangliosidoses

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 14, 2026

First Posted

October 21, 2026

Study Start (Estimated)

December 10, 2026

Primary Completion (Estimated)

April 1, 2030

Study Completion (Estimated)

April 1, 2030

Last Updated

October 21, 2026

Record last verified: 2026-10

Data Sharing

IPD Sharing
Will not share