Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
Starting soon · Phase 2
Conditions studied: Gaucher Disease Type 3
In brief
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).
Key facts
- Study ID
- NCT07715084
- Run by
- Azafaros B.V.
- People needed
- 20
- Starts
- 2026-09-28
- Expected to finish
- 2029-07-01
- Last updated by the study team
- 2026-07-20
Who can join
Age: 4 and older, up to 30. Sex: any. Healthy volunteers: not accepted.
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
Where it is running
- 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, Turkey (Türkiye)
- Gazi University Medical Faculty Hospital — Yenimahalle, Turkey (Türkiye)
Full record on ClinicalTrials.gov
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