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Starting soon Phase 2

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

NCT07715084 · tracked via the Priya Life Science Turkey tracker
Phase
Phase 2
Started
2026-09-28
Last updated
2026-07-20

Condition(s) studied

Gaucher Disease Type 3

Investigational drug(s) / intervention(s)

AZ-3102 →

AZ-3102: Daily oral intake of AZ-3102 orodispersible tablets

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).

Eligibility

Sex
ALL
Min age
4 Years
Max age
30 Years
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: * 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

Primary outcome measure(s)

  • Change in hemoglobin levels from Baseline — 24 weeks
  • Change in platelet count from Baseline — 24 weeks
  • Change in total spleen volume from Baseline — 24 weeks
    Assessed using magnetic resonance imaging (MRI)

Trial sites (7)

FacilityCityRegionStatus
SMS Medical College Jaipur Jaipur India
Maula a Azad medical college Delhi New Delhi India
Sir Ganga Ram Hospital New Delhi 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
Gazi University Medical Faculty Hospital Yenimahalle Turkey (Türkiye)
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07715084 on ClinicalTrials.gov ↗ ← All trials in Turkey