CARAT

• Research type

  Research Study

• Full title

  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

• IRAS ID

  1004202

• Contact name

  Veronica Baffa

• Contact email

  veronica.baffa@sanofi.com

• Sponsor organisation

  sanofi-aventis recherche et développement

• Eudract number

  2021-002320-20

• Clinicaltrials.gov Identifier

  NCT05280548

• Research summary

  The purpose of the study is to assess the effect of daily oral doses of the study drug called venglustat compared with standard of care therapies given for Fabry disease. This will be done by looking at the effect on heart function and what are the possible risks.

  The study will be with participants with Fabry disease who have an enlarged left ventricle of the heart and may or may not be receiving therapy for Fabry disease currently.

  Participation will be approximately 38 months at maximum including various regular tests & assessments such as MRI & ECHO.

• REC name

  East of England - Essex Research Ethics Committee

• REC reference

  22/EE/0002

• Date of REC Opinion

  21 Feb 2022

• REC opinion

  Further Information Favourable Opinion