NCT07715084

Brief Summary

A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).

Trial Health

67
Monitor

Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
34mo left

Started Sep 2026

Typical duration for phase_2

Geographic Reach
3 countries

7 active sites

Status
not yet recruiting

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 8, 2026

Completed
1 month until next milestone

First Posted

Study publicly available on registry

July 20, 2026

Completed
2 months until next milestone

Study Start

First participant enrolled

September 28, 2026

Expected
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2028

1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2029

Last Updated

July 20, 2026

Status Verified

July 1, 2026

Enrollment Period

1.3 years

First QC Date

June 8, 2026

Last Update Submit

July 15, 2026

Conditions

Gaucher Disease Type 3

Keywords

Nizubaglustat

Outcome Measures

Primary Outcomes (3)

  • Change in hemoglobin levels from Baseline

    24 weeks

  • Change in platelet count from Baseline

    24 weeks

  • Change in total spleen volume from Baseline

    Assessed using magnetic resonance imaging (MRI)

    24 weeks

Secondary Outcomes (8)

  • Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline

    24 weeks

  • Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline

    24 weeks

  • Clinical Global Impression of Change (CGI-C) score

    24 weeks

  • Participant/Caregiver Global Impression of Change (PGI-C) score

    24 weeks

  • Peak plasma concentration (Cmax) of Nizubaglustat

    24 weeks

  • +3 more secondary outcomes

Study Arms (1)

Nizubaglustat

EXPERIMENTAL

Participants will receive Nizubaglustat (AZ-3102) once daily during Study Period 1 (24 weeks), with a possible extension into Study Period 2 (72 weeks).

Drug: AZ-3102

Interventions

AZ-3102DRUG

Daily oral intake of AZ-3102 orodispersible tablets

Nizubaglustat

Eligibility Criteria

Age4 Years - 30 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Have a confirmed diagnosis of GD3 disease
  • Have at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening
  • Be aged 4-30 years old at the time of informed consent
  • Have the following symptoms of GD3 identified at Screening: GD-related anemia, thrombocytopenia, splenomegaly
  • Female participants of childbearing potential who are sexually active and willing to follow contraceptive guidance
  • Male participants with a female partner of childbearing potential who are willing to follow contraceptive guidance

You may not qualify if:

  • Any condition at Screening or Baseline that, in the opinion of the Principal Investigator, could interfere with study assessments
  • History of medical conditions other than GD3 that, in the opinion of the Principal Investigator, could confound scientific rigor or interpretation of results
  • Body weight \<10 kg at Screening
  • Presence of a neurologic disease other than GD3
  • Transfusion dependence
  • Prior splenectomy
  • The presence of severe renal impairment at Screening
  • Prior use of an investigational drug within 3 months before Screening
  • Prior participation in a clinical study involving gene therapy or stem cell transplantation
  • Current treatment, or treatment in the last 12 months before Screening, with enzyme replacement therapy (ERT) and/or substrate reduction therapy (SRT)
  • A positive serum pregnancy test (for women of childbearing potential)
  • Electrocardiogram (ECG) with an average Fridericia-corrected QT interval (QTcF) of \>450 msec for males and \>470 msec for females at Screening
  • Known allergies to azasugars or any study drug excipient

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

SMS Medical College Jaipur

Jaipur, 302004, India

Location

Maula a Azad medical college Delhi

New Delhi, 110002, India

Location

Sir Ganga Ram Hospital

New Delhi, 110060, India

Location

University of Child Health Sciences and the Children's Hospital

Lahore, Pakistan

Location

Clininal Trial Unit, Rehman Medical Institute

Peshawar, Pakistan

Location

Ege Universitesi Tip Fakultesi Hastanesi

Bornova, İzmir, 35100, Turkey (Türkiye)

Location

Gazi University Medical Faculty Hospital

Yenimahalle, 06560, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Central Study Contacts

Patient Advocacy Representative

CONTACT

Please reach out by emailinfo@azafaros.com

Contact for Healthcare Professionals

CONTACT

Please reach out by emailmedinfo@azafaros.com

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

June 8, 2026

First Posted

July 20, 2026

Study Start (Estimated)

September 28, 2026

Primary Completion (Estimated)

January 1, 2028

Study Completion (Estimated)

July 1, 2029

Last Updated

July 20, 2026

Record last verified: 2026-07

Data Sharing

IPD Sharing
Will not share

Locations

TU(2)IN(3)PA(2)