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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Daily oral intake of AZ-3102 orodispersible tablets
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
Change in hemoglobin levels from Baseline
Time frame: 24 weeks
Change in platelet count from Baseline
Time frame: 24 weeks
Change in total spleen volume from Baseline
Assessed using magnetic resonance imaging (MRI)
Time frame: 24 weeks
Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline
Scale ranges from 1 to 7, where a higher score equals a worse outcome.
Time frame: 24 weeks
Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline
Scale ranges from 1 to 7, where a higher score equals a worse outcome.
Time frame: 24 weeks
Clinical Global Impression of Change (CGI-C) score
Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.
Time frame: 24 weeks
Participant/Caregiver Global Impression of Change (PGI-C) score
Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.
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
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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)
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