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Title: Evaluating low-dose prednisone with standard therapy to improve heart function in patients with cardiac manifestations of Fabry disease

Fast replyRegistered drugNo placebo
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What is this trial about?

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

This clinical study focuses on patients with Fabry disease, a rare genetic condition that can affect the heart. The study will evaluate how adding low-dose prednisone (a corticosteroid medication) to standard treatments might help improve heart function in people who already receive either enzyme replacement therapy or chaperone therapy for their condition.

The research aims to determine if prednisone can reduce inflammation in the heart muscle, which is believed to be an important factor in heart problems caused by Fabry disease. Patients will receive either prednisone tablets or a placebo in addition to their regular Fabry disease treatment. The maximum daily dose of prednisone will be 5 milligrams, and the treatment period will last for 26 weeks.

During the study, doctors will monitor various aspects of heart health using different tests, including cardiac MRI (a detailed heart imaging test), echocardiography (heart ultrasound), and blood tests to measure substances that can indicate heart damage. They will also track how patients feel and how well they can perform daily activities to understand if the treatment is making a difference in their quality of life.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your participation begins with confirmation that you have Fabry disease and are currently receiving either enzyme replacement therapy or chaperone therapy

    Medical tests will confirm if you have heart-related symptoms of Fabry disease through heart scans, blood tests, and heart rhythm measurements

    A blood test will check your troponin T level, which must be above 14 ng/l to participate

  2. Step 2

    Treatment period start

    You will continue your current Fabry disease treatment (either enzyme replacement or chaperone therapy)

    You will receive prednisone (a steroid medication) taken by mouth in addition to your regular treatment

    The treatment aims to reduce inflammation in your heart muscle

  3. Step 3

    Monitoring and testing

    Regular blood tests to measure heart-related substances in your blood

    Heart scans including ultrasound and magnetic resonance imaging (MRI)

    Physical activity tests to measure your exercise capacity

    Quality of life questionnaires to assess your daily functioning

    Tests to check your kidney function

    Urine tests to measure substances related to Fabry disease

  4. Step 4

    Final assessment

    The main goal is to see if your troponin T levels decrease by half after completing the steroid treatment

    Final heart scans and blood tests will be performed

    The study will track any heart-related health events that occurred during your participation

Who can join the trial?

9 criteria

  • You must give written informed consent to participate in the study
  • You must be at least 18 years old when screening begins
  • You must be currently receiving treatment for Fabry disease with either:
    • Enzyme replacement therapy (agalsidase alfa or beta), or
    • Chaperone therapy (migalastat)
    • You must have heart problems related to Fabry disease, showing at least one of these signs:
      • Abnormal heart rhythm or electrical activity on ECG
      • Changes in heart structure visible on ultrasound (echocardiogram)
      • Abnormalities detected on cardiac MRI scan
      • You must have elevated levels of troponin T (a protein that indicates heart stress) above 14 ng/l
      • If you are a woman of childbearing age, you must be willing to use appropriate contraception during the study

Who cannot join the trial?

15 criteria

  • Active or chronic systemic infections (infections affecting multiple body systems)
  • Known hypersensitivity (allergic reaction) to prednisone or other corticosteroid medications
  • Presence of active peptic ulcer (open sores in stomach or small intestine)
  • Uncontrolled diabetes mellitus (high blood sugar levels)
  • Systemic fungal infections (fungal infections affecting multiple body parts)
  • Severe osteoporosis (significant bone weakness)
  • Presence of active malignancy (cancer)
  • Immunosuppression (weakened immune system) from any cause
  • Severe psychiatric disorders that could affect study participation
  • Participation in another clinical trial within the past 30 days
  • Pregnancy or breastfeeding
  • Inability to provide informed consent
  • History of poor compliance with medical treatments
  • Severe liver dysfunction (poor liver function)
  • Severe kidney dysfunction (poor kidney function)
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Investigated drugs

  • Prednisone

    is a corticosteroid medication that helps reduce inflammation in the body. In this trial, it is being studied as an additional treatment alongside standard therapies for Fabry disease. It works by suppressing the immune system and decreasing inflammation in the heart muscle.

  • Enzyme Replacement Therapy (ERT)

    is a standard treatment for Fabry disease that provides patients with a functioning version of the enzyme that their bodies are unable to produce naturally. This therapy helps break down certain fatty substances that would otherwise build up in the body's cells.

  • Chaperone Therapy (CHT)

    is another standard treatment for Fabry disease that works by helping to stabilize the patient's own defective enzymes. This therapy helps the body's natural enzymes work more effectively to break down harmful substances that accumulate in Fabry disease.

What is already known about the treatment

  • Prednisone

    A synthetic corticosteroid medication taken orally in tablet form that belongs to the glucocorticoid class of drugs. It works by suppressing the immune system and reducing inflammation throughout the body, making it particularly useful in treating various inflammatory conditions. In Fabry disease treatment, it is being studied as an adjunctive therapy alongside enzyme replacement therapy (ERT) or chaperone therapy (CHT) to potentially improve cardiac function by reducing myocardial inflammation. This well-established anti-inflammatory medication has been used in medicine for decades and continues to be a subject of clinical research for new therapeutic applications.

  • Enzyme Replacement Therapy (ERT)

    A treatment approach that involves intravenous administration of artificial enzymes to replace deficient or absent enzymes in patients with Fabry disease. This therapy works by providing functional copies of the alpha-galactosidase A enzyme, which helps break down specific fatty substances that would otherwise accumulate in blood vessels and organs. It represents a standard treatment option for Fabry disease, helping to manage symptoms and slow disease progression by addressing the underlying enzymatic deficiency.

  • Chaperone Therapy (CHT)

    An oral medication therapy that works by stabilizing the patient's own defective enzymes in Fabry disease, helping them to function more effectively. This treatment approach uses small molecules that act as pharmaceutical chaperones to help properly fold and transport the affected enzymes, thereby increasing their activity and reducing substrate accumulation. It offers an alternative treatment option for patients with specific mutations in the alpha-galactosidase A gene who are suitable for this approach.

Investigated diseases

Fabry Disease - A rare genetic disorder that affects the body's ability to break down specific types of fatty substances. The condition results from mutations in the GLA gene, which leads to the buildup of certain fats, primarily globotriaosylceramide (Gb3), in cells throughout the body. This accumulation causes progressive damage to tissues and organs, particularly affecting the heart, kidneys, and nervous system. The disease typically begins in childhood or adolescence and gradually worsens over time. Symptoms can include burning pain in hands and feet, decreased ability to sweat, dark skin spots, and gastrointestinal problems. As the disease progresses, it can lead to complications in various organs, particularly affecting heart function and kidney performance.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2025-521408-24-00Protocol codeSHIELD-FABRY/2025/1Estimated enrolment40 patientsSponsorMedical University Of Lodz

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