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)
Facility
City
Region
Status
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
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.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.