This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo-controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months. * Study visits will take place approximately every 3 months. * The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for an additional 12 months or until the Common Study End of Treatment Day (CSEOTD).
Double blind period: the total duration will be up to approximately of 14 months (1 month of screening 12 month of treatment period, and a possible follow-up period of 1 month if no participation in the open label extension period) Open-label extension period: the total duration will be approximately of 46 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 33 months), and 1 month of follow-up period)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
122
Pharmaceutical form: Tablet Route of administration: Oral
Pharmaceutical form: Tablet Route of administration: Oral
Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
Birmingham, Alabama, United States
UCLA Medical Center_Investigational Site Number: 8400006
Los Angeles, California, United States
University of California Irvine Medical Center- Site Number : 8400019
Orange, California, United States
Advent Health Orlando_Investigational Site Number: 8400008
Orlando, Florida, United States
Emory Genetics- Site Number : 8400010
Atlanta, Georgia, United States
Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain)
Time frame: From baseline to 6 months
Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain)
Time frame: From baseline to 12 months
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Time frame: From baseline to 6 month and 12 months
Frequency of rescue pain medication use
Number of days with use of rescue pain medications during the 6-month treatment period, divided by duration of the 6-month treatment period and multiplied by 100. The same definition will be used for the 12-month period.
Time frame: From baseline to 6 months and 12 months
Change in the percentage of days with at least 1 stool reflecting diarrhea (Bristol Stool Form Scale [BSFS] Type 6 or 7)
Time frame: From baseline to 6 month and 12 months
Change in tiredness component of FD-PRO
Time frame: From baseline to 6 month and 12 months
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO
Response is defined as at least a 30% decrease from baseline in the most bothersome of 3 FD-PRO items between neuropathic pain in upper extremities, neuropathic pain in lower extremities, and abdominal pain
Time frame: At 6 months and 12 months
Number of participants with adverse event (AE) and serious adverse event (SAE)
Time frame: From baseline to 6 month and 12 months
Change in the lens clarity (new or worsening lens opacities) by ophthalmological examination (by slit lamp exam at Visit 2 and Visit 6)
Time frame: From baseline to 12 months
Change in Beck Depression Inventory-II (BDI-II) score
Time frame: From baseline to 6 month and 12 months
Plasma venglustat concentrations at prespecified visits over the study duration
Time frame: From baseline to 6 month and 12 months
Maximum venglustat plasma concentration (Cmax)
Time frame: From baseline to 6 month and 12 months
Time to maximum venglustat plasma concentration (tmax)
Time frame: From baseline to 6 month and 12 months
Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
Time frame: From baseline to 6 month and 12 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Westchester Medical Center Healthcare Corporation- Site Number : 8400001
Hawthorne, New York, United States
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013
Cincinnati, Ohio, United States
Cleveland Clinic Site Number : 8400016
Cleveland, Ohio, United States
Children's Hospital Of Pittsburgh- Site Number : 8400009
Pittsburgh, Pennsylvania, United States
Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012
Dallas, Texas, United States
...and 48 more locations