Refer a Patient

Do You Have a Patient with Fabry Disease Who May Be Interested in Either the CARAT or PERIDOT Study?

If you have a patient with Fabry disease who is looking for an alternative oral treatment, or whose current treatment regimen is not controlling the progression of disease, the CARAT or PERIDOT study may be an option to improve symptoms.

About the CARAT Study

Study Purpose

The CARAT study is evaluating the safety and efficacy of a once-daily oral study drug, venglustat, compared to standard-of-care treatment (agalsidase beta or migalastat) on left ventricular hypertrophy (measured by left ventricular mass index) in patients with cardiac Fabry disease.

Study Design

This clinical research study is a phase 3, randomized, open-label, parallel-group clinical research study to evaluate the safety and efficacy of venglustat in the treatment of left ventricular hypertrophy in patients with Fabry disease. Participants will be randomized 1:1 to receive either venglustat or standard-of-care treatment.

Key Eligibility Criteria

Eligible participants:

  • Must be between 18 and 65 years old
  • Must have a confirmed diagnosis of Fabry disease
  • Must have Fabry-related heart involvement
  • Must have never received treatment or have gone without treatment with an approved or experimental therapy for Fabry disease for at least 6 months prior to screening.

Additional eligibility criteria may apply.

Study Participation

The total duration of the CARAT study for each participant can last approximately 3 years. This includes a 4-week screening period, an 18-month treatment period, and an 18-month long-term extension. During the long-term extension, all participants will receive the study drug. A follow-up visit will occur 30 days after the participant has received their final treatment dose.

About the PERIDOT Study

Study Purpose

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

Study Design

This clinical research study is a phase 3, randomized, double-blind, placebo-controlled clinical research study to evaluate the safety and efficacy of venglustat on reducing nerve and abdominal pain in patients with Fabry disease. Participants will be randomized 1:1 to receive either the study drug or the placebo.

Key Eligibility Criteria

Eligible participants:

  • Must be at least 18 years old
  • Must have a confirmed diagnosis of Fabry disease
  • Must have nerve pain or abdominal pain related to Fabry disease
  • Must have either never received Fabry disease treatment or not received treatment within the last 6 months

Additional eligibility criteria may apply.

Study Participation

The total duration of the PERIDOT study for each participant can last up to approximately 2 years. This includes a 4-week screening period, 12-month treatment period, and a 12-month open-label extension period where all the participants will receive the study drug. A follow-up visit will occur 30 days after the participant has received their final treatment dose.

Clinical Research Sites

If you have a patient who may be interested in participating in either the CARAT or PERIDOT study and who you think may prequalify, refer your patient to this study website to take the prescreening questionnaire to determine eligibility, or fill out a contact request form if you would like to request contact directly from an active clinical research site.

Find the Nearest Site

Enter your zip code below or click on the map to find the participating clinical research site(s) nearest you.

Find Sites For:

e.g., 12345

View full list of locations.

Active site locations

Future site locations