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Not yet recruitingNCT07715084Updated Jul 20, 2026

Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

A Phase 2 interventional study of AZ-3102 in Gaucher Disease Type 3, sponsored by Azafaros B.V.. Not yet recruiting at 7 sites in 3 countries. Open to participants aged 4 Years to 30 Years. Per ClinicalTrials.gov, last updated 2026-07-20.

Sponsored by Azafaros B.V. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
20
Allocation
Not applicable
Ages
4 Years to 30 Years
Sex
All
01

Study 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).

02

Conditions studied

  • Gaucher Disease Type 3

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Keywords

  • Nizubaglustat
03

Who can participate

Ages eligible
4 Years to 30 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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

Exclusion criteria

Exclusion Criteria:

  • 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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
20 participants (estimated)

Study arms

  • Experimental
    Nizubaglustat

    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

  • DrugAZ-3102

    Daily oral intake of AZ-3102 orodispersible tablets

05

What researchers measure

Primary outcomes

  1. Change in hemoglobin levels from Baseline

    Time frame: 24 weeks

  2. Change in platelet count from Baseline

    Time frame: 24 weeks

  3. Change in total spleen volume from Baseline

    Assessed using magnetic resonance imaging (MRI)

    Time frame: 24 weeks

Secondary outcomes

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

    Scale ranges from 1 to 7, where a higher score equals a worse outcome.

    Time frame: 24 weeks

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

    Scale ranges from 1 to 7, where a higher score equals a worse outcome.

    Time frame: 24 weeks

  3. Clinical Global Impression of Change (CGI-C) score

    Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.

    Time frame: 24 weeks

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

    Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.

    Time frame: 24 weeks

  5. Peak plasma concentration (Cmax) of Nizubaglustat

    Time frame: 24 weeks

  6. Area under the plasma concentration versus time curve (AUC0-24) for Nizubaglustat

    Time frame: 24 weeks

  7. Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylceramide (GlcCer) C16:0 and C18:0

    Time frame: 24 weeks

  8. Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylsphingosine (glcSph)

    Time frame: 24 weeks

06

Study locations

7 sites
  • SMS Medical College Jaipur
    Jaipur, 302004, India
  • Maula a Azad medical college Delhi
    New Delhi, 110002, India
  • Sir Ganga Ram Hospital
    New Delhi, 110060, India
  • University of Child Health Sciences and the Children's Hospital
    Lahore, Pakistan
  • Clininal Trial Unit, Rehman Medical Institute
    Peshawar, Pakistan
  • Ege Universitesi Tip Fakultesi Hastanesi
    Bornova, İzmir 35100, Turkey (Türkiye)
  • Gazi University Medical Faculty Hospital
    Yenimahalle, 06560, Turkey (Türkiye)
07

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07715084
Lead sponsor
Azafaros B.V.
Responsible party
Sponsor
First posted
Jul 20, 2026
Start date
Sep 28, 2026 (estimated)
Primary completion
Jan 2028 (estimated)
Completion
Jul 2029 (estimated)
Last update
Jul 20, 2026

Study contacts

Patient Advocacy Representative
Contact
info@azafaros.com
Please reach out by email
Contact for Healthcare Professionals
Contact
medinfo@azafaros.com
Please reach out by email

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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