A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and F… (NCT05280548) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
United States, Austria, Canada104 participantsStarted 2022-05-03
Plain-language 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, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.
* Study visits will take place approximately every 3 to 6 months
* Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.
Who can participate
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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 Criteria:
* 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)…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)