Fabry DiseasePhase 3PERIDOTNCT05206773
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
A Phase 3 study testing Venglustat (GZ402671) in people with Fabry Disease, run by Sanofi at 50 sites in 21 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Venglustat (GZ402671)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 122 participants
- Where
- 50 sitesin 21 countries
- Study dates
- 11 Mar 2022to 4 Jan 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female adult patients 16 year of age or older, who have had a previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease
- Patients who are treatment-naïve or without prior treatment with an approved or experimental therapy for Fabry disease within at least 6 months prior to screening.
- Average score of ≥3 (0=no symptom, 10=symptom as bad as you can imagine) 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.
- Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.
- Weight ≥30 Kg
- A signed informed consent must be provided prior to any study-related procedures.
Who cannot join
- Any manifestations of Fabry disease that preclude placebo administration.
- History of transient ischemic attack, stroke, myocardial infarction, heart failure, evidence of left ventricular hypertrophy and/or cardiac fibrosis, major cardiovascular surgery, or kidney transplantation.
- History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females.
- Patients with hepatitis C, HIV, or hepatitis B infection.
- Neuropathic pain in upper or lower extremities, or abdominal pain not related to Fabry disease.
- History of seizures currently requiring treatment.
Show all 18Show fewer
- Uncontrolled hypertension over the past 12 months prior to screening, or systolic BP >=150 or diastolic BP >=100 at screening.
- Estimated glomerular filtration rate <60 mL/min/1.73m².
- Urine protein to creatinine ratio >= 1 g/g at screening.
- Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II >28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit.
- Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID 19 requiring hospitalization within 6 months of enrollment.
- Moderate to severe hepatic impairment.
- History of drug and/or alcohol abuse.
- History of or active hepatobiliary disease.
- Liver enzymes (alanine aminotransferase (ALT)/aspartate aminotransferase (AST)) or total bilirubin >2 times the upper limit of normal (ULN).
- Initiation of chronic treatment for pain, or change in pain medication regimen, within 3 months prior to randomization.
- Strong or moderate inducers or inhibitors of cytochrome P450 3A within 14 days or 5 half-lives, whichever is longer, prior to randomization.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
50 sites in 21 countries.
50 sites in 21 countries
United States11 sites
Emory University School of Medicine - Atlanta- Site Number : 8400010
Atlanta, Georgia, 30322
University of Alabama -The Kirklin Clinic- Site Number : 8400011
Birmingham, Alabama, 35233
Cincinnati Children's Hospital Medical Center- Site Number : 8400013
Cincinnati, Ohio, 45229-3026
Renal Disease Research Institute - Dallas - Live Oak Street- Site Number : 8400012
Dallas, Texas, 75204
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004
Fairfax, Virginia, 22030
WMCHealth Advanced Physician Services- Site Number : 8400001
Hawthorne, New York, 10532
David Geffen School of Medicine at UCLA- Site Number : 8400006
Los Angeles, California, 90095
University of California Irvine Medical Center- Site Number : 8400019
Orange, California, 92868
AdventHealth Orlando- Site Number : 8400008
Orlando, Florida, 32803
UPMC Montefiore- Site Number : 8400009
Pittsburgh, Pennsylvania, 15213
University of Utah Health Hospital- Site Number : 8400005
Salt Lake City, Utah, 84132
China4 sites
Investigational Site Number : 1560001
Beijing, 100034
Investigational Site Number : 1560003
Shanghai, 200025
Investigational Site Number : 1560004
Taiyuan, 030001
Investigational Site Number : 1560006
Zhengzhou, 450052
Argentina3 sites
Investigational Site Number : 0320002
La Rioja, 5300
Investigational Site Number : 0320001
Pergamino, Buenos Aires, 2700
Investigational Site Number : 0320003
Quilmes, Buenos Aires, 1878
Canada3 sites
Investigational Site Number : 1240003
Calgary, Alberta, T2E 7Z4
Investigational Site Number : 1240001
Halifax, Nova Scotia, B3H 2Y9
Investigational Site Number : 1240005
Toronto, Ontario, M5T 3H7
Italy3 sites
Investigational Site Number : 3800005
Bologna, 40138
Investigational Site Number : 3800001
Naples, Napoli, 80131
Investigational Site Number : 3800004
Rome, Roma, 00168
Poland3 sites
Investigational Site Number : 6160002
Poznan, Greater Poland Voivodeship, 60-780
Investigational Site Number : 6160004
Rzeszów, Podkarpackie Voivodeship, 35-301
Investigational Site Number : 6160003
Wroclaw, Lower Silesian Voivodeship, 50-556
United Kingdom3 sites
Investigational Site Number : 8260001
Cambridge, Cambridgeshire, CB2 2QQ
Investigational Site Number : 8260002
London, England, NW3 2QG
Investigational Site Number : 8260003
Salford, Manchester, M6 8HD
Brazil2 sites
Hospital de Clinicas de Porto Alegre- Site Number : 0760001
Porto Alegre, Rio Grande do Sul, 90035903
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 0760002
São Paulo, 04020-041
Germany2 sites
Investigational Site Number : 2760003
Munich, 80337
Investigational Site Number : 2760001
Würzburg, 97080
Greece2 sites
Investigational Site Number : 3000003
Athens, 115 28
Investigational Site Number : 3000002
Ioannina, 455 00
Japan2 sites
Investigational Site Number : 3920004
Fukuoka, 814-0180
Investigational Site Number : 3920003
Tokyo, 105-8461
Mexico2 sites
Investigational Site Number : 4840002
Mexico City, 06700
Investigational Site Number : 4840001
Monterrey, Nuevo León, 64460
Turkey (Türkiye)2 sites
Investigational Site Number : 7920001
Ankara, 06560
Investigational Site Number : 7920002
İzmit, 41001
Australia1 site
Investigational Site Number : 0360001
Parkville, Victoria, 3050
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months
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).
Read the detailed description
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)
- Conditions
- Fabry Disease
- Being tested
- Venglustat (GZ402671), Placebo
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venglustat (GZ402671)Drug
Pharmaceutical form: Tablet Route of administration: Oral
PlaceboDrug
Pharmaceutical form: Tablet Route of administration: Oral
| Study group | Role | What they receive |
|---|---|---|
| Venglustat | Experimental | Participant will receive venglustat dose once daily for 12 months Receives: Drug: Venglustat (GZ402671) |
| Placebo | Placebo comparator | Participants will receive placebo once daily for 12 months Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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)
Measured: From baseline to 12 months
Secondary outcomes (12)
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Measured: 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.
Measured: 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)
Measured: From baseline to 6 month and 12 months
Change in tiredness component of FD-PRO
Measured: 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
Measured: At 6 months and 12 months
Number of participants with adverse event (AE) and serious adverse event (SAE)
Measured: 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)
Measured: From baseline to 12 months
Change in Beck Depression Inventory-II (BDI-II) score
Measured: From baseline to 6 month and 12 months
Plasma venglustat concentrations at prespecified visits over the study duration
Measured: From baseline to 6 month and 12 months
Maximum venglustat plasma concentration (Cmax)
Measured: From baseline to 6 month and 12 months
Time to maximum venglustat plasma concentration (tmax)
Measured: 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)
Measured: From baseline to 6 month and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2022
- Study start11 Mar 2022
- Primary completion17 Dec 2025
- Study completion4 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 122 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05206773
- Sponsor's protocol ID
- EFC17045
- Other IDs
- U1111-1256-9310, 2024-511990-31, 2021-002350-90
- Acronym
- PERIDOT
- Status last verified
- Oct 2026
- First submitted
- 20 Oct 2021
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months
- ClinicalTrials.gov ID
- NCT05206773
- Acronym / sponsor ID
- PERIDOT / EFC17045
- Conditions
- Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 122 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 11 Mar 2022 (actual)
- Primary completion
- 17 Dec 2025 (actual)
- Study completion
- 4 Jan 2027 (estimated)
- First posted
- 25 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Venglustat | Experimental | Drug: Venglustat (GZ402671)Participant will receive venglustat dose once daily for 12 months |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive placebo once daily for 12 months |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male and female adult patients 16 year of age or older, who have had a previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease
- Patients who are treatment-naïve or without prior treatment with an approved or experimental therapy for Fabry disease within at least 6 months prior to screening.
- Average score of ≥3 (0=no symptom, 10=symptom as bad as you can imagine) 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.
- Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.
- Weight ≥30 Kg
Key exclusion criteria (18 listed)
- Any manifestations of Fabry disease that preclude placebo administration.
- History of transient ischemic attack, stroke, myocardial infarction, heart failure, evidence of left ventricular hypertrophy and/or cardiac fibrosis, major cardiovascular surgery, or kidney transplantation.
- History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females.
- Patients with hepatitis C, HIV, or hepatitis B infection.
- Neuropathic pain in upper or lower extremities, or abdominal pain not related to Fabry disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Venglustat (GZ402671)Pharmaceutical form: Tablet Route of administration: Oral | Drug | Venglustat |
| PlaceboPharmaceutical form: Tablet Route of administration: Oral | Drug | Placebo |
Primary outcome
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
12 secondary outcomes
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Time frame: From baseline to 6 month and 12 months
Frequency of rescue pain medication use
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- EFC17045 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
50 sites in 21 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Venglustat (GZ402671) in people with Fabry Disease, run by Sanofi at 50 sites in 21 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, China, Argentina, Canada, Italy and 16 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.