Tests nizubaglustat in people with Gaucher disease type 3
Tests the drug nizubaglustat in people with Gaucher disease type 3.
Plain-language title generated from the registry record
Registry title: Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
Key facts
- Where
- Lahore and Peshawar, PakistanSee the 2 sites
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people aged 4-30 with a confirmed diagnosis of Gaucher disease type 3 (GD3) who have GD-related anemia, low platelets and an enlarged spleen. All join an open-label study of nizubaglustat (AZ-3102) lasting 96 weeks.
- Age
- 4-30 years old at the time of informed consent.
- Sex
- All sexes can join.
- Disease stage
- Confirmed GD3 with at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening. Symptoms at Screening: GD-related anemia, thrombocytopenia and splenomegaly.
- Earlier treatment
- Cannot have had enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) now or in the last 12 months before Screening. Cannot have had a splenectomy, an investigational drug in the last 3 months, or gene therapy or stem cell transplantation studies.
Main reasons for not taking part
- Body weight under 10 kg (22 lb) at Screening
- A neurologic disease other than GD3
- Transfusion dependence
- Severe renal impairment at Screening
- Known allergy to azasugars or any study drug excipient
Read by machine on 12 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 4 and 30 years old?
Source: NCT07715084, who can take part
Where in Pakistan
- University of Child Health Sciences and the Children's HospitalLahore, PakistanContact: Patient Advocacy RepresentativeNo longer taking part
- Clininal Trial Unit, Rehman Medical InstitutePeshawar, PakistanContact: Patient Advocacy RepresentativeNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Azafaros B.V.
- Trial code
- NCT07715084
- Record updated
- 12 November 2026
- Original description
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).Read full original descriptionShow less
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)
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).
Inclusion: 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: Prior splenectomy; 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); 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; Presence of a neurologic disease other than GD3; The presence of severe renal impairment at Screening; Known allergies to azasugars or any study drug excipient; 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; Body weight <10 kg at Screening; Transfusion dependence.