Table of Contents
- Trial overview
- Who can join
- What is being measured
- Treatments in the study
- Study design and phase
- Patient terms to know
Trial overview
The available trial for AGALSIDASE BETA is an interventional Phase 3 study in adults with Fabry disease.[1] It is authorised and plans to include 110 participants.[1]
The study is designed to compare the effect of venglustat tablets with standard of care Fabry therapies on heart size changes over 18 months in people with Fabry disease and left ventricular hypertrophy.[1]
Who can join
The trial includes male and female adult participants with Fabry disease.[1] It also focuses on people who already have left ventricular hypertrophy, which means thickening of the heart muscle in the main pumping chamber.[1]
No other participation details are provided in the trial data, so the main known target group is adults with Fabry disease and this heart finding.[1]
What is being measured
The main endpoint is the slope of left ventricular mass index measured by cardiac magnetic resonance imaging, also called cardiac MRI.[1] In simple words, the study tracks how the heart muscle measurement changes over time.[1]
This is a heart-focused outcome, so the trial is not just looking at whether treatment is given, but whether there is a measurable change in heart structure during the study period.[1]
Treatments in the study
The trial data list several study treatments and comparators, including AGALSIDASE BETA as part of standard Fabry therapy options, along with Replagal, Fabrazyme, Galafold, and venglustat.[1] The brief summary says the study compares venglustat with standard of care Fabry therapies.[1]
The source does not provide a detailed treatment plan beyond the listed interventions, so the key point is that the study is comparing treatment approaches in Fabry disease.[1]
Study design and phase
This is an interventional study, which means researchers assign treatments and then measure the results.[1] It is in Phase 3, a later stage of clinical testing that usually involves more participants and compares treatment effects in a larger group.[1]
The study status is listed as Authorised, and the planned enrollment is 110 people.[1]
Patient terms to know
Fabry disease is the main condition being studied in this trial.[1] Left ventricular hypertrophy means the heart muscle is thicker than normal in the left pumping chamber.[1]
Left ventricular mass index is a measurement used to describe the amount of heart muscle, adjusted for body size.[1] Central reading means the MRI scans are reviewed in one central place to keep the measurements consistent.[1]
