Venglustat for Fabry Disease
(PERIDOT Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing a medication called venglustat to see if it can help reduce pain in people with Fabry disease. The medication works by targeting the root causes of nerve and abdominal pain.
Do I have to stop taking my current medications for the trial?
The trial does not specify if you must stop all current medications, but you cannot have started or changed chronic pain treatment within 3 months before joining. Also, you can't use strong or moderate inducers or inhibitors of cytochrome P450 3A within 14 days before joining.
Will I have to stop taking my current medications?
The trial does not specify if you must stop taking your current medications, but you should not have started or changed any chronic pain treatment within 3 months before joining. Also, you cannot use certain medications that affect liver enzymes within 14 days before starting the trial.
What safety data is available for Venglustat in treating Fabry Disease?
Is the drug Venglustat a promising treatment for Fabry Disease?
How does the drug Venglustat differ from other treatments for Fabry disease?
Venglustat is unique because it is an oral medication that works by inhibiting glucosylceramide synthase, which reduces the production of glycosphingolipids that accumulate in Fabry disease. This approach is different from enzyme replacement therapies that are typically administered intravenously and focus on replacing the deficient enzyme in patients.678910
What data supports the idea that Venglustat for Fabry Disease is an effective drug?
Who Is on the Research Team?
Clinical Sciences & Operations
Principal Investigator
Sanofi
Are You a Good Fit for This Trial?
Adults with Fabry disease experiencing neuropathic or abdominal pain can join this trial if they haven't had treatments for Fabry in the last 6 months. They must have a confirmed diagnosis, be at least 18 years old, and agree to use double contraception methods. Exclusions include recent changes in pain meds, certain cardiovascular issues, uncontrolled hypertension, severe liver problems, active infections like COVID-19 within specific timeframes.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either venglustat or placebo for 12 months to evaluate its effect on neuropathic and abdominal pain
Follow-up
Participants are monitored for safety and effectiveness after the double-blind treatment period
Open-label extension
Participants who completed the double-blind period may receive venglustat for up to an additional 12 months, with further treatment until a common study end date
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- Venglustat (GZ402671)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sanofi
Lead Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris