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A Study of Acoramidis for Long-Term Safety in Patients with Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy

Verified siteInvestigationalNo placebo
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What is this trial about?

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

This study involves people with Transthyretin Amyloidosis, a condition where abnormal protein deposits build up in the heart muscle, causing the heart to work less effectively. This particular study focuses on a form of the condition that affects the heart, called variant transthyretin amyloid cardiomyopathy, which has been newly diagnosed in participants. The treatment being studied is Acoramidis, also known by its code name AG10, which is given as a tablet taken by mouth.

The purpose of this study is to evaluate the long-term safety and how well people tolerate acoramidis when taken over an extended period. This is an extension study, which means it continues treatment for people who have already completed an earlier study called ACT-EARLY. Participants who finished that previous study within the past 60 days and who still have the diagnosis can join this extension study to continue receiving the medication.

During the study, participants will take acoramidis tablets regularly, and doctors will monitor them for any side effects or unwanted reactions to the medication. The study will track various health measures including heart function through ultrasound images of the heart, blood tests that measure substances related to heart stress and damage, heart rhythm patterns, and overall health status. Doctors will also watch for any hospitalizations related to heart problems, changes in symptoms, development of heart rhythm disturbances, and other health conditions that can occur with this disease. The study will continue for several years to gather information about the long-term effects of the medication.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and consent

    This extension study begins after completion of the previous study called AG10-501 (also known as ACT-EARLY). Entry into this extension study must occur within 60 days of finishing the previous study.

    Before any procedures begin, a signed informed consent form must be provided, confirming the willingness to participate in this extension study.

    The diagnosis of transthyretin amyloid cardiomyopathy (a condition where abnormal protein deposits affect the heart) must have been confirmed during the previous study.

  2. Step 2

    Contraception requirements

    If female and capable of becoming pregnant, a highly effective method of contraception must be used starting from the first day of the study and continuing for 30 days after the last dose of the study medication.

    If male and sexually active with a female partner of childbearing potential, a double-barrier method of birth control must be used during the study and for 30 days after the last dose of the study medication. Sperm donation must be avoided for at least 30 days after the last dose.

  3. Step 3

    Treatment with acoramidis

    The study medication is called acoramidis (also referred to as AG10). The active ingredient is acoramidis hydrochloride.

    The medication is provided as a tablet that is taken by mouth.

    The study is designed as an open-label study, which means both the patient and the medical team will know that acoramidis is being administered.

    The specific dosage, frequency, and exact duration of treatment are determined by the study protocol and will be explained by the study team.

  4. Step 4

    Safety monitoring visits

    Throughout the study, regular visits will be scheduled to monitor safety and how well the medication is tolerated.

    During these visits, physical examinations will be performed to check for any abnormal findings that may be clinically relevant.

    Vital signs (such as blood pressure, heart rate, and temperature) will be measured and reviewed for any abnormal changes.

    Electrocardiograms (ECG, a test that records the electrical activity of the heart) will be performed to detect any abnormal heart rhythm or conduction problems.

    Laboratory tests will be conducted using blood samples to check for changes in safety parameters that may require attention.

    Any adverse events (unwanted or harmful reactions) and serious adverse events (severe reactions that may require hospitalization or are life-threatening) will be recorded and evaluated.

  5. Step 5

    Assessment of heart function and structure

    Echocardiography (an ultrasound test of the heart) will be performed at scheduled intervals to measure changes in the structure and function of the heart muscle.

    Strain parameters (measurements that show how well the heart muscle is contracting) will also be assessed during these imaging tests.

    Blood tests will measure levels of NT-proBNP (a substance released when the heart is under stress) and high-sensitivity troponin I (a marker of heart muscle damage). Changes in these levels from the beginning of the study will be tracked.

  6. Step 6

    Monitoring for disease progression and complications

    The development of symptomatic disease (when symptoms of the heart condition become noticeable) will be monitored throughout the study.

    Any episodes of heart failure will be recorded, including hospitalizations or unplanned visits related to worsening heart function.

    New heart rhythm problems (arrhythmias) will be monitored, including new occurrences of irregular heartbeats such as atrial fibrillation or atrial flutter, dangerous fast heartbeats from the lower chambers of the heart, or problems with the heart's electrical conduction that may require a pacemaker.

    New diagnoses or surgeries related to conditions that may be associated with the disease will be tracked. These include carpal tunnel syndrome (compression of a nerve in the wrist), tendon ruptures, spinal narrowing, joint problems in the hips or knees, finger stiffness, or shoulder joint issues.

    Hospitalizations related to heart and blood vessel problems, as well as deaths from any cause or from heart-related causes, will be recorded.

  7. Step 7

    Study completion

    The extension study is planned to continue until approximately April 2032.

    Participation may be discontinued earlier if adverse events occur that require stopping the medication, or if withdrawal from the study is requested.

    After the last dose of study medication, contraception requirements must continue for an additional 30 days.

Who can join the trial?

5 criteria

  • You must have completed the AG10-501 study (also called the ACT-EARLY study) within the past 60 days with a diagnosis of ATTR-CM, which is a heart condition caused by abnormal protein deposits in the heart muscle
  • You must be willing and able to provide signed informed consent, which means you agree in writing to participate in the study after understanding what it involves. This must be done before any study procedures begin
  • If you are a woman who can become pregnant and have sexual intercourse with men, you must agree to use a highly effective method of contraception (birth control) from the first day of the study and for 30 days after taking the last dose of the study medication
  • If you are a man who has not had a vasectomy (a surgical procedure to prevent pregnancy) and have sexual intercourse with women who can become pregnant, you must agree to use a double-barrier method of birth control (such as a condom combined with another form of contraception) during the study and for 30 days after taking the last dose of the study medication
  • If you are a man, you must agree not to donate sperm for at least 30 days after taking the last dose of the study medication

Who cannot join the trial?

1 criterion

  • No exclusion criteria have been provided in the available study information.
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Investigated drugs

Acoramidis is a medication being tested in this study for people with a heart condition called transthyretin amyloid cardiomyopathy. This is a disease where abnormal protein builds up in the heart muscle, making it harder for the heart to work properly. Acoramidis is designed to help stabilize the protein that causes this buildup, which may slow down or prevent further damage to the heart. In this trial, researchers want to see how safe the medication is when taken over a long period of time and whether people can tolerate it well.

What is already known about the treatment

Acoramidis – Acoramidis is an investigational medication being studied in tablet form taken by mouth for the treatment of transthyretin amyloid cardiomyopathy, a rare disease where abnormal protein deposits damage the heart muscle. The drug works at the molecular level by binding to and stabilizing the transthyretin protein in the blood, preventing it from breaking apart and forming harmful deposits in heart tissue. It belongs to a class of drugs called transthyretin stabilizers, which are designed to slow or stop the progression of this serious heart condition. This medication is currently in Phase 3 clinical trials to evaluate its long-term safety and effectiveness in patients with newly diagnosed forms of the disease.

Investigated diseases

Transthyretin Amyloidosis – A rare disease in which abnormal deposits of a protein called transthyretin build up in various organs and tissues throughout the body. The protein misfolds and accumulates, forming clumps called amyloid fibrils that interfere with normal organ function. When these deposits affect the heart, the condition is called transthyretin amyloid cardiomyopathy, which causes the heart muscle to become stiff and thick. This stiffening makes it difficult for the heart to pump blood effectively and fill properly between beats. The disease can also affect nerves, leading to problems with sensation, movement, and automatic body functions. Over time, the progressive accumulation of amyloid deposits causes worsening symptoms and organ dysfunction.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513676-18-00Protocol codeAG10-504Estimated enrolment279 patientsSponsorEidos Therapeutics Inc.

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