A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-509715-91-00

Acronym

EFC16158 - CARAT

Enrollment

Registered

2024-06-20

Start date

2022-05-06

Completion date

Unknown

Last updated

2025-11-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry’s disease

Brief summary

Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)

Detailed description

Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation, Change in T1 relaxation time, measured by cardiac MRI (central reading), Change in global longitudinal strain, measured by echocardiography (central reading), Percent Change in tiredness component of FDPRO, Percent Change in swelling in lower extremities component of FD-PRO, Number of participants with adverse event (AE) and serious adverse event (SAE), Change in Beck Depression Inventory-II (BDI-II) score, Change in the lens clarity by ophthalmological examination, Plasma venglustat concentrations at prespecified visits over the study duration

Interventions

DRUGReplagal 1 mg/ml concentrate for solution for infusion.

DRUGvenglustat GZ402671 - SAR402671

DRUGGalafold 123 mg hard capsules

DRUGFabrazyme 35 mg powder for concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)	—

Secondary

Measure	Time frame
Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation, Change in T1 relaxation time, measured by cardiac MRI (central reading), Change in global longitudinal strain, measured by echocardiography (central reading), Percent Change in tiredness component of FDPRO, Percent Change in swelling in lower extremities component of FD-PRO, Number of participants with adverse event (AE) and serious adverse event (SAE), Change in Beck Depression Inventory-II (BDI-II) score, Change in the lens clarity by ophthalmological examination, Plasma venglustat concentrations at prespecified visits over the study duration	—

Measure

Time frame

—

Countries

Austria, Czechia, Denmark, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026