Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study comparing venglustat tablets with standard therapy for adult patients with Fabry disease and left ventricular hypertrophy

Fast replyRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on patients with Fabry disease, a rare genetic condition that affects various organs in the body. The research aims to evaluate a new medication called venglustat compared to standard treatments including agalsidase alfa, agalsidase beta, and migalastat in treating patients who have an enlarged heart muscle, also known as left ventricular hypertrophy.

The study will compare two groups of patients over 18 months. One group will receive venglustat tablets taken by mouth, while the other group will continue their current standard treatment. The standard treatments are given either as an infusion into a vein or as oral capsules, depending on the specific medication.

Throughout the study, patients will undergo various medical examinations including cardiac MRI scans to measure heart muscle size, heart function tests, kidney function tests, and assessments of various symptoms related to Fabry disease. The study will also monitor the safety of the treatment by checking for any side effects that may occur during the treatment period.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment and group assignment

    Your participation begins after signing the informed consent form

    You will be randomly assigned to one of two groups: either receiving venglustat tablets or continuing your current standard treatment (if any)

    Your current treatment may include agalsidase alfa, agalsidase beta, or migalastat

  2. Step 2

    Treatment period

    The study will last for 18 months

    If assigned to venglustat group: you will receive venglustat tablets for oral use

    If assigned to standard care group: you will continue receiving your current medication through either oral capsules or intravenous infusion, depending on your treatment

  3. Step 3

    Regular monitoring

    Your heart will be examined using cardiac magnetic resonance imaging (MRI)

    Regular heart ultrasound (echocardiography) will be performed

    Your kidney function will be monitored through blood tests

    Eye examinations will check your lens clarity

    You will complete questionnaires about tiredness and mood changes

    Blood samples will be taken to measure medication levels in your body

  4. Step 4

    Safety monitoring

    Any side effects or health changes will be recorded throughout the study

    Regular check-ups will monitor your overall health

    The study will track changes in symptoms related to Fabry disease, including tiredness and leg swelling

  5. Step 5

    Study completion

    Final assessments will be conducted at the end of the 18-month period

    All test results will be compared with your initial measurements

    The main focus will be on changes in your heart muscle thickness as measured by MRI

Who can join the trial?

8 criteria

  • Age between 18 and 65 years old
  • Must have a confirmed diagnosis of Fabry disease and previous symptoms related to this condition
  • Can be either currently receiving treatment with agalsidase alfa, agalsidase beta, or migalastat (medications used to treat Fabry disease), or not receiving any treatment
  • Must have left ventricular hypertrophy (a condition where the heart's main pumping chamber becomes thickened)
  • If female, must not be pregnant or breastfeeding
  • Must use appropriate contraception methods during the study
  • If male, must not donate sperm during the study period
  • Must be willing and able to sign an informed consent form before any study procedures begin

Who cannot join the trial?

14 criteria

  • Age below 18 years or above 65 years
  • Patients with severe heart conditions other than those related to Fabry disease
  • Pregnant or breastfeeding women
  • Patients currently participating in other clinical trials
  • Patients with severe kidney dysfunction (poor kidney function)
  • History of allergic reactions to similar medications
  • Patients who cannot follow the study protocol due to physical or mental conditions
  • Patients taking medications that could interfere with the study drug
  • Patients with uncontrolled high blood pressure
  • Inability to provide informed consent
  • History of substance abuse within the past year
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
  • Patients who have had major surgery within the last 3 months
  • Severe liver problems or abnormal liver function tests
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Venglustat

    is an investigational oral medication being studied for treating Fabry disease. It works by reducing the buildup of harmful substances in the body's cells, particularly in the heart. This medication aims to help patients with Fabry disease who have developed heart problems, specifically left ventricular hypertrophy (enlarged heart muscle).

  • Standard of care Fabry therapies

    refers to the currently approved and commonly used treatments for Fabry disease. These treatments help manage the symptoms and complications of Fabry disease by replacing the missing enzyme or reducing the accumulation of harmful substances in the body's tissues and organs.

What is already known about the treatment

Venglustat - An oral medication being investigated in clinical trials for the treatment of Fabry disease, specifically targeting patients with left ventricular hypertrophy. This drug belongs to the class of glucosylceramide synthase inhibitors, working at the molecular level by reducing the accumulation of harmful lipids in various tissues and organs, particularly in the heart. Venglustat represents a potential new therapeutic approach that differs from traditional enzyme replacement therapies commonly used in Fabry disease management, and is being studied in comparison with standard care treatments to evaluate its effectiveness in reducing left ventricular mass index in affected patients.

Investigated diseases

Fabry Disease - A rare genetic disorder that affects the body's ability to break down specific types of fats called glycolipids. The disease causes these fatty substances to build up in blood vessels, tissues, and organs throughout the body. This accumulation typically begins in childhood and gradually increases over time, affecting multiple body systems. The condition is inherited and primarily affects the heart, kidneys, nervous system, and skin. As the disease progresses, it can lead to decreased function in affected organs and tissues. The disease affects males more severely than females, though both can develop symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-509715-91-00Protocol codeEFC16158 - CARATEstimated enrolment110 patientsSponsorSanofi-Aventis Recherche & Developpement

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.