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Venglustat

Clinical trials of Venglustat are studying whether it can help people with Fabry disease and Gaucher disease type 3. These studies look at safety, effectiveness, and patient-reported symptoms in adults and some adolescents. The trials compare Venglustat with standard treatments or placebo in different patient groups.

Table of Contents

Clinical trial overview

The source data includes four interventional studies of Venglustat, all in rare inherited diseases.[1] Two studies are in Fabry disease, and two studies are in Gaucher disease type 3.[1] The trials are in Phase 2 and Phase 3, which means they are testing both early signals and larger confirmatory results.[1]

These studies are designed to measure safety, symptom changes, and disease-related markers.[1] The main patient groups are adults with Fabry disease, adults with Gaucher disease type 3, and in one study, adolescents with Gaucher disease type 3.[1]

Fabry disease: heart study

NCT05280548 is a Phase 3 study in male and female adults with Fabry disease.[1] It is authorised and plans to enroll 110 participants.[1] The study compares Venglustat with standard Fabry treatments, including Replagal, Fabrazyme, and Galafold.[1]

The main outcome is the slope of left ventricular mass index, measured by cardiac MRI with central reading.[1] In simple terms, this looks at how the size or thickness of the left side of the heart changes over time.[1] The brief summary says the goal is to compare the effect of Venglustat with standard Fabry therapies on left ventricular mass index over 18 months in participants with Fabry disease and left ventricular hypertrophy.[1]

Fabry disease: pain study

NCT05206773 is another Phase 3 study in male and female participants aged 16 years and older with Fabry disease.[2] It is authorised and plans to enroll 124 participants.[2] This study compares Venglustat with placebo tablets.[2]

The main outcomes are the percent change from baseline at 6 months and 12 months in the most bothersome symptom from three patient-reported Fabry disease items: neuropathic pain in the upper limbs, neuropathic pain in the lower limbs, or abdominal pain.[2] The brief summary says the study is for participants who are treatment-naïve or who have not received treatment for at least 6 months before screening.[2] Patient-reported outcome means the patient reports the symptom directly, rather than the doctor measuring it only from tests.[2]

Gaucher disease type 3: Phase 3 study

NCT05222906 is a Phase 3 study in adult and pediatric patients with Gaucher disease type 3.[3] It is authorised and plans to enroll 50 participants.[3] The study compares Venglustat with Cerezyme and includes placebo tablets and placebo for Cerezyme in the treatment plan.[3]

The main outcomes are changes in the Scale for the Assessment and Rating of Ataxia (SARA) modified total score and the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score.[3] SARA is a measure of balance and coordination problems, while RBANS is a set of tests that checks thinking and memory skills.[3] The brief summary says the study is evaluating efficacy in adults and in pediatric patients aged 12 to under 18 years with Gaucher disease type 3.[3]

Gaucher disease type 3: Phase 2 study

NCT02843035 is a Phase 2 study in adult patients with Gaucher disease type 3, with a monotherapy extension and some information also linked to Gaucher disease type 1 in the condition list.[4] It is completed and enrolled 18 participants.[4] The study used Venglustat alone and in combination with Cerezyme.[4]

The primary outcomes included the number of participants with treatment-emergent adverse events and the levels of the biomarkers lyso-GL1 and GL-1 in cerebrospinal fluid.[4] The brief summary explains that Part 1 screened and evaluated CSF biomarkers to help distinguish Gaucher disease type 3 from Gaucher disease type 1.[4] Parts 2 and 3 looked at short-term and long-term safety and tolerability of Venglustat with Cerezyme, and Part 4 looked at safety and tolerability of Venglustat monotherapy in adults who stayed systemically stable on the combination treatment.[4]

What the trial outcomes mean

These studies measure different kinds of outcomes because Fabry disease and Gaucher disease type 3 can affect different body systems.[1][3] In the Fabry heart study, the focus is on heart structure over time.[1] In the Fabry pain study, the focus is on symptoms that patients feel in daily life.[2]

In the Gaucher disease type 3 studies, the focus includes safety, biomarkers, movement, and cognition.[3][4] Biomarkers are measurable substances that can help show how a disease is behaving.[4] Together, these outcomes help researchers learn whether Venglustat may be useful for specific rare disease problems in the studied groups.[1][2][3][4]

Trial ID Phase Condition studied Status Enrollment
NCT05280548 Phase 3 Fabry disease Authorised 110
NCT05206773 Phase 3 Fabry disease Authorised 124
NCT05222906 Phase 3 Gaucher disease type 3 Authorised 50
NCT02843035 Phase 2 Gaucher disease type 3, Gaucher disease type 1 Completed 18

FAQ

  • What conditions are being studied in Venglustat trials? The trials are studying Fabry disease and Gaucher disease type 3. One study also includes adult and pediatric patients with Gaucher disease type 3.
  • Who can take part in these studies? Some studies include adult men and women, while one Fabry study includes participants age 16 years and older. One Gaucher disease type 3 study includes adults and children ages 12 to under 18 years.
  • What are the main goals of the Venglustat trials? The main goals are to check whether Venglustat is safe and whether it helps with disease-related problems. The studies also measure pain, heart changes, ataxia, and cognition.
  • What phases are the Venglustat trials in? The trials in the source data are Phase 2 and Phase 3 studies. Phase 2 studies usually look at early safety and activity, while Phase 3 studies compare treatment effects in larger groups.
  • What kinds of results are measured in these trials? The trials measure heart muscle changes, pain scores, adverse events, biomarker levels in spinal fluid, ataxia scores, and cognitive test results. These outcomes help show whether treatment may improve symptoms or disease markers.

Ongoing Clinical Trials on Venglustat

  • 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

    Not recruiting

    3 1
    Investigated drugs:
    Austria Denmark Finland France Germany Greece +4

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

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Czechia Denmark France Germany Greece +5

  • Study on the Effectiveness and Safety of Venglustat for Adults and Children with Gaucher Disease Type 3

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy

  • Study of Venglustat and Imiglucerase for Adult Patients with Gaucher Disease Type 3

    Not recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

Glossary

  • Fabry disease: A rare inherited disease that can affect many parts of the body, including the heart and nerves.
  • Gaucher disease type 3: A rare inherited disease that can affect the brain, nerves, and other organs.
  • Phase 2: An early clinical trial phase that helps researchers learn more about safety and whether a treatment shows signs of benefit.
  • Phase 3: A later trial phase that usually compares a treatment with standard care or placebo in larger groups of people.
  • Interventional study: A study where people receive a treatment or placebo so researchers can measure its effects.
  • Placebo: An inactive treatment that looks like the study medicine, used for comparison.
  • Primary outcome: The main result a study is designed to measure.
  • Left ventricular mass index: A measure of the size or thickness of the left side of the heart.
  • Cardiac magnetic resonance imaging (MRI): A scan that uses magnets to create detailed pictures of the heart.
  • Treatment-emergent adverse events (TEAEs): Health problems that start or get worse after a person begins treatment in a study.
  • Biomarker: A measurable substance in the body that can help show how a disease is behaving.
  • Ataxia: Problems with balance and coordination.
  • Cognition: Thinking skills such as memory, attention, and understanding.

References