A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease (PERIDOT)
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|ClinicalTrials.gov Identifier: NCT05206773|
Recruitment Status : Recruiting
First Posted : January 25, 2022
Last Update Posted : February 21, 2023
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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 adult participants 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.
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: Venglustat (GZ402671) Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||114 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Masking Description:||Participants enrolled in the open-label extension will be treated with venglustat only.|
|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 Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months|
|Actual Study Start Date :||March 11, 2022|
|Estimated Primary Completion Date :||November 30, 2024|
|Estimated Study Completion Date :||January 6, 2026|
Participant will receive venglustat dose once daily up to 12 months
Drug: Venglustat (GZ402671)
Pharmaceutical form: Tablet Route of administration: Oral
Placebo Comparator: Placebo
Participants will receive placebo once daily up to 12 months
Pharmaceutical form: Tablet Route of administration: Oral
- 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) [ 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, and abdominal pain) [ Time Frame: From baseline to 12 months ]
- 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 ]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.
- 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 ]
- Percent 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 ]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
- 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 ]
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Male and female adult patients 18 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.
- Elevated plasma globotriaosylsphingosine (lysoGL3) at screening, or a previous biopsy of any organ that shows deposition of GL3.
- 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.
- A signed informed consent must be provided prior to any study-related procedures.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05206773
|Contact: Trial Transparency email recommended (Toll free for US & Canada)||800-633-1610 ext option 6||Contact-US@sanofi.com|
|Study Director:||Clinical Sciences & Operations||Sanofi|
|Responsible Party:||Genzyme, a Sanofi Company|
|Other Study ID Numbers:||
2021-002350-90 ( EudraCT Number )
U1111-1256-9310 ( Registry Identifier: ICTRP )
|First Posted:||January 25, 2022 Key Record Dates|
|Last Update Posted:||February 21, 2023|
|Last Verified:||February 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||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|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
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