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AGALSIDASE ALFA

Clinical trials investigating AGALSIDASE ALFA are studying treatment in people with Fabry disease, including adults with heart involvement. These trials look at how well study treatments work and how safe they are, with a focus on heart measures such as left ventricular mass index. The target group in the available trial is male and female adults with Fabry disease and left ventricular hypertrophy.

Table of contents

Trial overview

The available study is NCT05280548, an interventional Phase 3 trial in adults with Fabry disease.[1] It is authorised and plans to enroll 110 participants.[1] The study is designed to compare the effect of venglustat with standard of care Fabry therapies on heart changes over 18 months.[1]

Who can participate

The trial includes male and female adult participants with Fabry disease.[1] It specifically targets people with left ventricular hypertrophy, which means thickening of the main pumping chamber of the heart.[1]

  • Adults only: the source data says the study is for adult participants.[1]
  • Both men and women: the study includes male and female participants.[1]
  • Fabry disease with heart involvement: the study focuses on people who already have left ventricular hypertrophy.[1]

What is being measured

The main outcome is the slope of left ventricular mass index measured by cardiac magnetic resonance imaging, also called cardiac MRI.[1] In simple terms, the study checks how the thickness or size of the heart muscle changes over time.[1] The MRI images are read centrally, which means the scans are reviewed in one main place to keep the measurements consistent.[1]

The study follows participants over 18 months.[1] This time frame helps researchers see whether the heart measure improves, stays stable, or gets worse during the study period.[1]

Treatments and comparators

The trial compares venglustat with standard of care Fabry therapies.[1] The source data lists several treatment options used in the study context, including Replagal, Fabrazyme, and Galafold, along with venglustat.[1]

  • Replagal is listed as an infusion treatment given through a vein.[1]
  • Fabrazyme is also listed as an infusion treatment.[1]
  • Galafold is listed as an oral treatment, which means taken by mouth.[1]
  • venglustat is listed as an oral treatment in this study.[1]

The trial title and summary show that the main goal is to compare study treatment with the usual Fabry treatments used in practice.[1]

Trial phase and size

This is a Phase 3 study, which usually means the treatment is being tested in a larger group and compared with other available options.[1] The planned enrollment is 110 participants.[1] The study status is Authorised.[1]

What the trial means for patients

This trial is focused on a heart-related problem in Fabry disease, not on the whole disease picture.[1] The researchers are mainly asking whether the study treatment can change left ventricular mass index over time compared with standard Fabry therapies.[1] Because the study includes adults of both sexes with heart muscle thickening, it is aimed at a specific patient group rather than all people with Fabry disease.[1]

Trial ID Phase Condition studied Status Enrollment
NCT05280548 Phase 3 Fabry disease Authorised 110

FAQ

  • What is being studied in AGALSIDASE ALFA clinical trials? The available trial studies treatment effects in Fabry disease, especially changes in heart structure. It compares study treatments with standard care options used in Fabry disease.
  • Who can take part in the trial? The trial includes male and female adult participants with Fabry disease. It also focuses on people with left ventricular hypertrophy, which means thickening of the heart muscle.
  • What phase is the trial? The trial is a Phase 3 study. Phase 3 trials are larger studies that compare how well treatments work and continue to assess safety.
  • What condition is the trial for? The trial is for Fabry disease, also called Fabry’s disease in the source data. It focuses on people with heart involvement.
  • What outcome is the trial measuring? The main outcome is the slope of left ventricular mass index measured by cardiac MRI. This shows how the heart muscle changes over time during the study.

Ongoing Clinical Trials on AGALSIDASE ALFA

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

    Not recruiting

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

Glossary

  • Fabry disease: A rare inherited disease studied in this trial. The trial focuses on adults with this condition.
  • Left ventricular mass index: A measurement of the size or thickness of the left side of the heart muscle, adjusted for body size.
  • Left ventricular hypertrophy: Thickening of the heart muscle on the left side of the heart. This is one of the key features being studied.
  • Cardiac MRI: A heart scan that uses magnetic fields to make detailed pictures of the heart. In this trial, it is used to measure heart muscle changes.
  • Central reading: A way of checking scan results in one main place so the measurements are more consistent.
  • Phase 3: A later stage of clinical testing where treatments are compared in larger groups of people.
  • Interventional study: A study where participants receive a treatment or intervention and researchers measure the effects.
  • Standard of care: The usual treatments already used for a disease. The trial compares study treatment against these options.
  • Oral use: Taken by mouth, such as tablets or capsules.
  • Intravenous use: Given through a vein, usually by infusion.

References