Not yet recruiting NCT07715084

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

Phase II Interventional Gaucher Disease Type 3

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: AZ-3102.
Who it may be relevant to: Registry conditions: Gaucher Disease Type 3. Basic parameters: 4 years — 30 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: India, Pakistan, Turkey (Türkiye)
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Official title

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).

Secondary outcome measures (8)

Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline [Time frame: 24 weeks]
Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline [Time frame: 24 weeks]
Clinical Global Impression of Change (CGI-C) score [Time frame: 24 weeks]
Participant/Caregiver Global Impression of Change (PGI-C) score [Time frame: 24 weeks]
Peak plasma concentration (Cmax) of Nizubaglustat [Time frame: 24 weeks]
Area under the plasma concentration versus time curve (AUC0-24) for Nizubaglustat [Time frame: 24 weeks]
Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylceramide (GlcCer) C16:0 and C18:0 [Time frame: 24 weeks]
Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylsphingosine (glcSph) [Time frame: 24 weeks]

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

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

India · 3 centers

Pakistan · 2 centers

Turkey (Türkiye) · 2 centers

NCT: NCT07715084 · AZA-001-202-GD3