Study Details
Testing a new drug called Venglustat to treat nerve pain and abdominal pain in adults with Fabry Disease (The PERIDOT (EFC17045) Study)
(IRB#: IRB_00153570)
This study will test a new drug called Venglustat for treating nerve pain and abdominal pain in adults with Fabry Disease. This study is for people who have not been treated by enzyme replacement therapy or who stopped taking enzyme replacement therapy for at least 6 months. Participants in this study will be randomized to be treated with Venglustat or a placebo. Later in the study, participants on the placebo can be switched to Venglustat. Participants will be in the study for 2 years and 2 months. There will be several clinic visits for the study and there may be the option for some home health visits. There will also be phone call visits.
- All genders
- Over 18 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: Over 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Be at least 18 years old
- Have a previous confirmed diagnosis of Fabry disease
- Have nerve pain or abdominal pain related to Fabry disease
- Have either never received Fabry disease treatment or have not received treatment within the last six months
Will I be paid for my time?
Yes
IRB#: IRB_00153570
PI: Nicola Longo
Department: PEDIATRIC GENETICS
Approval Date: 2023-08-09 06:00:00
Specialties: Pediatric Genetics