Clinical Trials in Turkey / NCT07715084
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
Condition(s) studied
Gaucher Disease Type 3
Investigational drug(s) / intervention(s)
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
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 | 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) |
More Azafaros B.V. trials in Turkey
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