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A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
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 up to an additional 12 months.
Study details:
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 31 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 18 months), and 1 month of follow-up period).
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 16 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2022-03-11
Primary completion: 2024-11-30
Study completion finish: 2025-10-06
Study type
TREATMENT
Phase
PHASE3
Trial ID
NCT05206773
Intervention or treatment
DRUG: Venglustat (GZ402671)
DRUG: Placebo
Conditions
- • Fabry Disease
Find a site
Closest Location:
Investigational Site Number : 0360001
Research sites nearby
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Investigational Site Number : 0360001
Parkville, Victoria, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Venglustat
| DRUG: Venglustat (GZ402671)
|
PLACEBO_COMPARATOR: Placebo
| DRUG: Placebo
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
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, and abdominal pain) | Not Specified | 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, and abdominal pain) | Not Specified | From baseline to 12 months |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Percent change in plasma globotriaosylsphingosine (lyso-GL-3) | Not Specified | 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. | 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) | Not Specified | From baseline to 6 month and 12 months |
Percent change in tiredness component of FD-PRO | Not Specified | 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 | At 6 months and 12 months |
Number of participants with adverse event (AE) and serious adverse event (SAE) | Not Specified | 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) | Not Specified | From baseline to 12 months |
Change in Beck Depression Inventory-II (BDI-II) score | Not Specified | From baseline to 6 month and 12 months |
Plasma venglustat concentrations at prespecified visits over the study duration | Not Specified | From baseline to 6 month and 12 months |
Maximum venglustat plasma concentration (Cmax) | Not Specified | From baseline to 6 month and 12 months |
Time to maximum venglustat plasma concentration (tmax) | Not Specified | 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) | Not Specified | From baseline to 6 month and 12 months |
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