Status:
ACTIVE_NOT_RECRUITING
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Lead Sponsor:
Sanofi
Conditions:
Fabry Disease
Eligibility:
All Genders
18-65 years
Phase:
PHASE3
Brief Summary
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, aga...
Detailed Description
Randomized period: the total duration will be up to approximately of 20 months (1 month screening 18 months of treatment and a possible follow-up period of 1 month if no participation in the long-term...
Eligibility Criteria
Inclusion
- Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease.
- Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated.
- Left ventricular hypertrophy.
- Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant.
- A signed informed consent must be provided prior to any study-related procedures.
Exclusion
- History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation.
- History of seizures currently requiring treatment.
- Underlying medical condition that may cause or contribute to left ventricular hypertrophy.
- Asymmetric hypertrophy by cardiac MRI at screening if considered by central reader to be not related to Fabry disease.
- Advanced cardiac fibrosis, defined as significant late gadolinium enhancement affecting 3 or more segments involving \>50% of myocardial thickness on screening cardiac MRI.
- 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.
- Estimated glomerular filtration rate \<45 mL/min/1.73m2.
- 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.
- Patients with hepatitis C, HIV, or hepatitis B infection.
- Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment.
- History of drug and/or alcohol abuse.
- Moderate to severe hepatic impairment.
- History of or active hepatobiliary disease.
- Liver enzymes (alanine aminotransferase/aspartate aminotransferase) or total bilirubin \>2 times the upper limit of normal.
- Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to randomization.
- Known contraindication to undergoing MRI or known hypersensitivity to gadolinium-based contrast agents.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Key Trial Info
Start Date :
May 3 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 6 2027
Estimated Enrollment :
104 Patients enrolled
Trial Details
Trial ID
NCT05280548
Start Date
May 3 2022
End Date
December 6 2027
Last Update
January 13 2025
Active Locations (54)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Alabama -The Kirklin Clinic- Site Number : 8400010
Birmingham, Alabama, United States, 35233
2
University of California Los Angeles Medical Center- Site Number : 8400008
Los Angeles, California, United States, 90095
3
Emory University School of Medicine - Atlanta- Site Number : 8400009
Atlanta, Georgia, United States, 30322
4
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005
Chicago, Illinois, United States, 60611