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Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven't received treatment for 6 months

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What is this trial about?

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

This clinical trial focuses on Fabry Disease, a rare genetic condition that affects multiple parts of the body and causes various symptoms including nerve pain and stomach pain. The study tests a medication called venglustat (also known as GZ402671 or SAR402671) that is taken as tablets by mouth. Some participants will receive the actual medication while others will receive placebo tablets.

The purpose of this research is to determine how well venglustat works in treating nerve pain and abdominal pain in people with Fabry Disease who have not received previous treatment or have not been treated for at least 6 months. The study will last for 12 months, during which participants will take either venglustat tablets or placebo tablets daily.

Throughout the study, participants will be monitored for changes in their pain levels and other symptoms related to Fabry Disease. The medication will be tested at different doses (up to 15 mg per day) to evaluate its effectiveness and safety. Various aspects of the disease will be tracked, including changes in specific substances in the blood, use of pain medications, and other symptoms such as tiredness and digestive problems.

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 screening and eligibility confirmation

    You will undergo screening to confirm your Fabry disease diagnosis

    Your pain levels will be evaluated using a scale from 0 (no symptom) to 10 (worst possible symptom)

    Basic health checks will be performed, including weight measurement (minimum 30 kg required)

    An eye examination will be conducted to assess your lens clarity

  2. Step 2

    Treatment assignment

    You will be randomly assigned to receive either venglustat tablets or placebo tablets

    Neither you nor your doctor will know which treatment you are receiving

    The tablets are to be taken by mouth

  3. Step 3

    6-month evaluation period

    Your most troublesome symptom (upper body nerve pain, lower body nerve pain, or stomach pain) will be monitored

    Blood tests will measure specific substances related to Fabry disease

    Your use of additional pain medication will be recorded

    Your tiredness levels and digestive symptoms will be tracked

    Regular safety checks will be performed

  4. Step 4

    12-month evaluation period

    Continued monitoring of your pain symptoms

    Additional eye examination to check for any changes in lens clarity

    Assessment of your mood using a specialized questionnaire

    Blood tests to measure medication levels

    Monitoring of any side effects or health changes

  5. Step 5

    Safety monitoring

    Throughout the study, you will be monitored for any side effects

    Regular health assessments will be conducted

    Any new or worsening symptoms will be recorded

Who can join the trial?

8 criteria

  • Must be at least 16 years old with a confirmed diagnosis of Fabry disease
  • Must be either: - Never treated for Fabry disease before, or - Not received any treatment (approved or experimental) for at least 6 months before screening
  • Must experience significant pain symptoms, scoring 3 or higher on a scale from 0-10 in at least one of these areas: - Nerve pain in arms - Nerve pain in legs - Stomach pain
  • Must weigh at least 30 kilograms (approximately 66 pounds)
  • Must agree to use two forms of contraception during the study period
  • For female participants: - Must not be pregnant - Must not be breastfeeding
  • For male participants: - Must not donate sperm during the study period
  • Must sign an informed consent form before any study procedures begin

Who cannot join the trial?

14 criteria

  • Age below 16 years
  • Currently receiving enzyme replacement therapy (ERT) or chaperone therapy for Fabry disease
  • Treatment with any Fabry disease therapy within 6 months before screening
  • History of severe allergic reactions to medications
  • Pregnant women or women planning pregnancy during the study period
  • Breastfeeding women
  • Severe kidney problems (defined as glomerular filtration rate below 30 mL/min/1.73m²)
  • Significant liver disease or abnormal liver function tests
  • Unstable heart conditions
  • Active cancer or history of cancer within the past 5 years
  • Participation in another clinical trial within 30 days before this study
  • Mental conditions that could interfere with study participation
  • Drug or alcohol abuse within the past year
  • Any condition that, in the investigator's opinion, makes the person unsuitable for the study
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Investigated drugs

Venglustat is an experimental medication being studied for treating Fabry disease. It aims to help reduce neuropathic pain (nerve pain) and abdominal pain in patients with this rare genetic condition. The medication works by targeting the underlying cause of Fabry disease by reducing the buildup of harmful substances in the body's cells. This is an oral medication taken by patients who either have never received treatment for Fabry disease before or haven't been treated for at least 6 months.

What is already known about the treatment

Venglustat - An oral medication being investigated in clinical trials for the treatment of Fabry Disease, a rare genetic disorder affecting the body's ability to break down certain types of fats. This novel drug works by inhibiting the production of glycosphingolipids, which are fatty substances that accumulate in various organs and tissues in patients with Fabry Disease. Venglustat belongs to the class of substrate reduction therapy medications and is specifically designed to reduce the neuropathic and abdominal pain associated with the condition. The drug is administered in pill form and is being evaluated in Phase 3 clinical trials for its effectiveness in treating patients aged 16 and older who are either treatment-naïve or have been untreated for at least 6 months.

Investigated diseases

Fabry Disease - A rare genetic disorder that affects the body's ability to break down specific types of fats called globotriaosylceramide (GL-3). These fats gradually accumulate in blood vessels, tissues, and organs throughout the body. The condition typically begins in childhood or adolescence and can cause various symptoms including burning pain in hands and feet, decreased ability to sweat, dark skin spots, and digestive problems including abdominal pain and diarrhea. As the disease progresses, it can affect multiple body systems, particularly impacting the heart, kidneys, and nervous system. The disease is inherited in an X-linked pattern, meaning it primarily affects males, though females can also develop symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511990-31-00Protocol codeEFC17045 - PERIDOTEstimated enrolment124 patientsSponsorSanofi-Aventis Recherche & Developpement

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