A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

Studying the Effect of an Investigational Medication on Heart Health in Fabry Disease

Recruiting
18 years - 65 years
All
Phase 3
90 participants needed
8 Locations

Study Overview

This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.

  • Study visits will take place approximately every 3 to 6 months
  • Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 34 months with the total study duration up to 4.4 years maximum.

Study Details

Randomized period: the total duration will be up to approximately of 20 months (1 month screening 18 months of treatment and a possible follow-up period of 1 month if no participation in the long-term extension period)

Long-term extension period: the total duration will be from minimum 19 months (18 months of treatment and 1 month of follow-up period) to maximum 35 months (34 months of treatment and 1 month of follow-up period). The maximum total study duration is approximately 4.4 years

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Fabry Disease
  • Age: 18 years - 65 years
  • Gender: All

Inclusion Criteria:

  • Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease.
  • Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated.
  • Left ventricular hypertrophy.
  • Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant.
  • A signed informed consent must be provided prior to any study-related procedures.

Exclusion Criteria:

  • History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation.
  • History of seizures currently requiring treatment.
  • Underlying medical condition that may cause or contribute to left ventricular hypertrophy.
  • Asymmetric hypertrophy by cardiac MRI at screening if considered by central reader to be not related to Fabry disease.
  • Advanced cardiac fibrosis, defined as significant late gadolinium enhancement affecting 3 or more segments involving >50% of myocardial thickness on screening cardiac MRI.
  • History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females.
  • Estimated glomerular filtration rate <45 mL/min/1.73m2.
  • Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II >28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit.
  • Patients with hepatitis C, HIV, or hepatitis B infection.
  • Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment.
  • History of drug and/or alcohol abuse.
  • Moderate to severe hepatic impairment.
  • History of or active hepatobiliary disease.
  • Liver enzymes (alanine aminotransferase/aspartate aminotransferase) or total bilirubin >2 times the upper limit of normal.
  • Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to randomization.
  • Known contraindication to undergoing MRI or known hypersensitivity to gadolinium-based contrast agents.
        The above information is not intended to contain all considerations relevant to a potential
        participation in a clinical trial.

Updated on 29 May 2024. Study ID: NCT05280548

This study investigates the effect of an investigational medication on heart health in people with Fabry disease. Fabry disease is a genetic disorder that affects the body's ability to break down a certain type of fat, leading to a buildup that can cause various symptoms, including heart problems. The study will focus on adults who have left ventricular hypertrophy, a condition where the heart's left ventricle is thickened.

Participants will be randomly assigned to receive either the investigational medication or standard care. Standard care may include treatments like agalsidase alfa, agalsidase beta, or migalastat. The study will monitor changes in the left ventricular mass index, which measures the size of the heart's left ventricle. Participants might undergo regular medical assessments to track their heart health.

  • Who can participate: Adults aged 18 to 65 with Fabry disease and left ventricular hypertrophy can participate. Key exclusion criteria include a history of significant heart conditions or other medical issues.
  • Study details: Participants will receive either the investigational medication or standard care, which may include treatments like agalsidase alfa, agalsidase beta, or migalastat. Regular assessments will monitor heart health.
  • Study Timelines: The study will last 20 months.

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