Purpose of the CARAT Study

The CARAT study is evaluating the safety and efficacy of a once-daily oral study drug, venglustat, compared to standard-of-care treatment on left ventricular hypertrophy in patients with Fabry-related heart involvement.

Who May Qualify

Approximately 90 patients, males and females, are expected to participate in the CARAT study globally. You may be eligible for this study if you:

  • Are between 18 and 65 years old
  • Have a confirmed diagnosis of Fabry disease
  • Have Fabry-related heart involvement

You may also be currently receiving treatment with an approved therapy for Fabry disease (agalsidase beta or migalastat) or may be currently untreated.

You will NOT be eligible if you:

  • Have severe depression or a history of an untreated, unstable major affective disorder within one year of the screening visit
  • Have advanced cardiac fibrosis or asymmetric hypertrophy
  • Have advanced kidney disease

Additional criteria will be assessed by a study doctor to confirm your eligibility.

Study Participation

The total length of the CARAT study can last approximately up to three years. This includes a four-week screening period to make sure the clinical research study is a good match for you, an 18-month treatment period in which you will receive either the study drug or standard-of-care therapy (agalsidase beta or migalastat), and an 18-month long-term extension study in which all participants will receive venglustat. A follow-up visit will occur 30 days after you have received the final treatment dose.

Throughout the clinical research study, your study doctor may perform a number of tests and procedures, including but not limited to:

  • Recording your medical history
  • Conducting a physical exam
  • Measuring your vital signs
  • Conducting a pregnancy test (if applicable)
  • Collecting blood and urine samples
See if you may prequalify

Purpose of the PERIDOT Study

The PERIDOT study is evaluating the safety and efficacy of a once-daily oral study drug on reducing nerve pain and abdominal pain in Fabry disease.

Who May Qualify

Approximately 114 patients, males and females, are expected to participate in the PERIDOT study globally. You may be eligible for this study if you:

  • Are at least 18 years old
  • Have a confirmed diagnosis of Fabry disease
  • Have nerve pain or abdominal pain related to Fabry disease
  • Are treatment-na├»ve or without prior treatment with an approved or experimental therapy for Fabry disease within at least six months prior to screening

You will NOT be eligible if you:

  • Have severe depression or a history of an untreated, unstable major affective disorder within one year of the screening visit
  • Have advanced kidney, heart, or cerebrovascular disease

Additional criteria will be assessed by a study doctor to confirm your eligibility.

Study Participation

The total length of the PERIDOT study can last up to approximately two years. This includes a four-week screening period to make sure the clinical research study is a good match for you, a 12-month treatment period in which you will receive either the study drug or the placebo, and a 12-month open-label extension, where all participants will receive the study drug. A follow-up visit will occur 30 days after you have received the final treatment dose. A placebo is a substance that looks just like the study drug but contains no active ingredients.

Throughout the clinical research study, your study doctor may perform a number of tests and procedures, including but not limited to:

  • Recording your medical history
  • Conducting a physical exam
  • Measuring your vital signs
  • Conducting a pregnancy test (if applicable)
  • Collecting blood and urine samples

You will also be required to complete a daily electronic diary.

See if you may prequalify

About the Study Drug

Venglustat, the study drug being researched in the CARAT and PERIDOT studies, is a potential oral therapy that is not dependent on the type of Fabry mutation.

Key characteristics:

  • Works by inhibiting glucosylceramide synthase and reduces the production of glycosphingolipids
  • Established treatment target in lysosomal storage diseases
  • Oral investigational compound for all Fabry subtypes
  • Potential to penetrate tissues affected by Fabry disease