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Study on the Effects of Tafamidis in Patients with Transthyretin Cardiac Amyloidosis

Verified siteRegistered 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 trial is focused on studying a condition known as transthyretin cardiac amyloidosis, a disease where abnormal proteins build up in the heart, affecting its function. The treatment being tested is a medication called tafamidis, also known by its code name FX-1006. Tafamidis is provided in the form of soft capsules and is taken orally. The purpose of the study is to observe changes in the heart using magnetic resonance imaging (MRI) before and after treatment with tafamidis over a period of 12 months.

Participants in the study will receive either tafamidis or a placebo. Throughout the study, the heart's structure and function will be monitored using MRI to assess any changes. The study will also track the frequency of hospitalizations related to heart failure and overall survival rates. Additionally, changes in heart-related symptoms and physical activity levels will be evaluated using questionnaires and walking tests.

The study aims to provide a better understanding of how tafamidis affects the heart in patients with transthyretin cardiac amyloidosis. By comparing MRI results and other health indicators before and after treatment, researchers hope to gain insights into the potential benefits of tafamidis for managing this condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will begin participation in a clinical trial focused on evaluating the impact of tafamidis on cardiac amyloidosis.

    The study involves the use of Vyndaqel 61 mg soft capsules, which are taken orally.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to establish baseline measurements. This includes cardiac magnetic resonance imaging (MRI) to evaluate heart function and structure.

    The assessment will measure parameters such as ventricular volumes, mass, ejection fraction, and other specific imaging markers.

  3. Step 3

    Medication administration

    The patient will begin taking tafamidis at a dosage of 61 mg once daily. This medication is administered in the form of a soft capsule.

    The treatment duration is set for 12 months, during which the patient will continue to take the medication as prescribed.

  4. Step 4

    Ongoing monitoring

    Throughout the trial, regular follow-up visits will be scheduled to monitor the patient's health and response to the medication.

    These visits will include repeated cardiac MRI scans to track changes in heart function and structure over time.

  5. Step 5

    Evaluation of outcomes

    The primary outcome of the study is to observe changes in cardiac MRI parameters from the baseline at various points during the trial.

    Secondary outcomes include monitoring the frequency of heart failure-related hospitalizations, overall health status, and changes in specific heart function tests.

  6. Step 6

    Completion of the study

    At the end of the 12-month treatment period, a final assessment will be conducted to evaluate the overall impact of tafamidis on the patient's cardiac health.

    The study is expected to conclude by July 2026, with all data collected and analyzed to determine the effectiveness of the treatment.

Who can join the trial?

9 criteria

  • Must have a specific heart condition called transthyretin cardiac amyloidosis.
  • Must have had a special heart scan called diphosphonate scintigraphy showing certain levels of uptake, and heart involvement confirmed by an echocardiogram (a heart ultrasound) with a heart wall thickness greater than 12 mm, and no signs of a specific protein in the blood or urine.
  • Alternatively, must have a positive biopsy (a small tissue sample) from outside the heart showing ATTR amyloidosis and heart involvement confirmed by an echocardiogram with a heart wall thickness greater than 12 mm.
  • Or, must have a positive heart biopsy for ATTR amyloidosis.
  • Must have a history of heart failure classified as NYHA I-III, which means mild to moderate heart failure.
  • Must be between 18 and 90 years old at the time of screening.
  • Must be able to provide written consent and be willing to follow the study requirements.
  • Patients with or without kidney failure can participate, but those with severe kidney failure (stage 4/5) will not have a specific heart test called late gadolinium enhancement.
  • Must be able to follow the treatment guidelines for a medication called tafamidis at a dose of 61 mg.

Who cannot join the trial?

4 criteria

  • Patients who are not male cannot participate in the study.
  • Patients who are not within the specified age range cannot participate. The age range is not specified here, but it is important for eligibility.
  • Patients with medical conditions other than Transthyretin cardiac amyloidosis may not be eligible. This is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, are not included in this study.
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Investigated drugs

Tafamidis is a medication used in this clinical trial to treat cardiac amyloidosis. The trial aims to assess how the heart's condition changes with the use of tafamidis over a period of 12 months. Cardiac amyloidosis is a disease where abnormal protein deposits in the heart, and tafamidis works by stabilizing these proteins to prevent further damage to the heart.

What is already known about the treatment

Tafamidis – Tafamidis is administered orally in capsule form. It is currently recognized in medical literature as a treatment for transthyretin cardiac amyloidosis, a condition where abnormal protein deposits affect the heart. The main therapeutic indication for Tafamidis is to stabilize the transthyretin protein, preventing it from breaking apart and forming amyloid deposits. At the molecular level, Tafamidis binds to the transthyretin protein, stabilizing its structure and preventing the formation of amyloid fibrils. It is classified pharmacologically as a transthyretin stabilizer.

Investigated diseases

Transthyretin Cardiac Amyloidosis – This is a condition where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits can cause the heart walls to become stiff, affecting the heart's ability to pump blood effectively. Over time, this can lead to symptoms such as fatigue, shortness of breath, and swelling in the legs. The disease progresses as more amyloid builds up, further impairing heart function. It is considered a rare disease and can significantly impact a person's quality of life. The progression of the disease can vary, with some individuals experiencing a gradual worsening of symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-518708-29-00Protocol codeMGNT2022Estimated enrolment30 patientsSponsorUnidade Local De Saude De Tras-Os-Montes E Alto Douro E.P.E.

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