Last updated 46 days ago
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
114 patients around the world
Available in Argentina, United States
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 31 months (12
month of OLE treatment, additional OLE treatment until a common study end of treatment
date (CSEOTD, approximately 18 months), and 1 month of follow-up period)
Genzyme, a Sanofi Company
3Research sites
114Patients around the world
Requirements for the patient
From 16 Years
All Gender
Medical requirements
- 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 diseasePatients 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.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.
Sites
Fundación Cori para la Investigación y Prevención del Cáncer - La Rioja
Recruiting
Instituto de Investigaciones Clínicas Quilmes
Recruiting
Instituto de Nefrología Pergamino SRL
Recruiting
StudyPERIDOT
SponsorGenzyme, a Sanofi Company
Study typeInterventional
Requirements
From 16 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT05206773
