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Substrate Reduction Therapy
Venglustat for Fabry Disease (PERIDOT Trial)
Phase 3
Recruiting
Research Sponsored by Sanofi
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male and female adult patients 16 years of age or older, with a previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease
Average score of ≥3 on the participant-defined most-bothersome symptom (among neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain), as measured by the Fabry Disease Patient-Reported Outcome (FD-PRO) at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 6 months and 12 months
Awards & highlights
PERIDOT Trial Summary
This trial will study the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in adults who have not been treated or have been treated for less than 6 months.
Who is the study for?
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.Check my eligibility
What is being tested?
The study is testing Venglustat tablets against placebo over a year to see if they reduce neuropathic and abdominal pain in adults with Fabry disease. Participants will either get the real drug or a dummy pill without knowing which one. After this 'blind' phase, there's an extra year where everyone gets Venglustat.See study design
What are the potential side effects?
While not specified here, potential side effects of Venglustat may include typical drug reactions such as digestive discomforts (nausea/vomiting), headaches, dizziness or fatigue. Since it's under study its full range of side effects are still being evaluated.
PERIDOT Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 16 or older with a confirmed diagnosis and symptoms of Fabry disease.
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My worst symptom from Fabry disease scores 3 or more on the FD-PRO.
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I weigh 30 kg or more.
PERIDOT Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at 6 months and 12 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 6 months and 12 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
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)
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)
Secondary outcome measures
Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
Change in Beck Depression Inventory-II (BDI-II) score
Change in the lens clarity (new or worsening lens opacities) by ophthalmological examination (by slit lamp exam at Visit 2 and Visit 6)
+9 morePERIDOT Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: VenglustatExperimental Treatment1 Intervention
Participant will receive venglustat dose once daily up to 12 months
Group II: PlaceboPlacebo Group1 Intervention
Participants will receive placebo once daily up to 12 months
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Venglustat (GZ402671)
2023
Completed Phase 1
~30
Find a Location
Who is running the clinical trial?
SanofiLead Sponsor
2,164 Previous Clinical Trials
3,514,866 Total Patients Enrolled
13 Trials studying Fabry Disease
2,145 Patients Enrolled for Fabry Disease
Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,516 Total Patients Enrolled
25 Trials studying Fabry Disease
14,661 Patients Enrolled for Fabry Disease
Clinical Sciences & OperationsStudy DirectorSanofi
862 Previous Clinical Trials
2,019,725 Total Patients Enrolled
6 Trials studying Fabry Disease
74 Patients Enrolled for Fabry Disease
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My blood pressure has been stable and below 150/100 for the past year.My liver does not work well.I have a history of liver or bile duct disease.I haven't started or changed pain medication in the last 3 months.I am 16 or older with a confirmed diagnosis and symptoms of Fabry disease.I have severe depression or an untreated mood disorder in the past year.I have hepatitis C, HIV, or hepatitis B.I haven't taken strong or moderate drugs that affect liver enzymes in the last 14 days or more.I am currently being treated for seizures.My worst symptom from Fabry disease scores 3 or more on the FD-PRO.I have nerve pain in my arms, legs, or stomach not due to Fabry disease.I weigh 30 kg or more.I have a heart rhythm problem but it's been stable for over a year.I am using two forms of birth control and am not pregnant or breastfeeding.I haven't received any Fabry disease treatment in the last 6 months.My kidney function is reduced.My Fabry disease symptoms are too severe for a placebo.I have a history of heart or major cardiovascular issues, or kidney transplantation.I tested positive for COVID-19 recently or was hospitalized for it in the last 6 months.
Research Study Groups:
This trial has the following groups:- Group 1: Venglustat
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is Venglustat's (GZ402671) official regulatory status?
"Venglustat's (GZ402671) safety is estimated to be a 3. This Phase 3 rating means that, while there isn't extensive evidence of its efficacy, multiple rounds of testing have proven it to be safe."
Answered by AI
Are there many hospitals conducting this trial in Canada?
"Currently, there are 7 clinical trial sites operational. These locations include Advent Health Orlando 301 East Princeton St_Site Number: 8400008 in Orlando, University of Manitoba, Children's Hospital Research Institute of Manitoba, Room 511C John Buhler Research Center - site number 1240002 in Winnipeg, and Oregon Health and Sciences University - site number 8400007 in Portland."
Answered by AI
What is the projected number of people who will be taking part in this research project?
"In order to move this study forward, 114 individuals who match the pre-determined criteria must participate. Those willing and eligible can enroll at either Advent Health Orlando or University of Manitoba."
Answered by AI
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