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Study on the Safety of Acoramidis in Patients with Transthyretin Amyloid Cardiomyopathy Who Completed a Previous Trial

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 clinical trial is focused on studying a condition known as Transthyretin Amyloid Cardiomyopathy (ATTR-CM). This is a heart disease where abnormal proteins build up in the heart, affecting its function. The study involves a treatment called Acoramidis (AG10), which is taken in tablet form. The purpose of the study is to monitor the safety and how well patients tolerate this medication.

Participants in this study are those who have already completed a previous trial involving the same medication. During the study, participants will continue to take Acoramidis and will be regularly monitored for any side effects or changes in their health. This includes checking for any serious health events, changes in physical exams, vital signs, and heart tests like electrocardiograms (ECG). The study will also look at overall health outcomes, such as any changes in walking distance and heart-related hospital visits.

The study aims to provide valuable information on the long-term safety of Acoramidis for people with ATTR-CM. It will help understand how the medication affects the heart and overall health over time. Participants will be closely observed to ensure their safety and to gather data that could benefit future patients with this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing 30 months of the previous study, AG10-301, including all assessments and procedures.

    A written informed consent form must be understood and signed before starting any study procedures.

  2. Step 2

    Medication administration

    The medication used in this study is acoramidis (AG10), administered in the form of a tablet.

    The route of administration is oral use.

  3. Step 3

    Safety and monitoring

    The main objective is to assess the safety and tolerability of acoramidis in participants with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM).

    Safety parameters include monitoring for serious adverse events, adverse events leading to treatment discontinuation, and any abnormal findings in physical examinations, vital signs, and electrocardiogram (ECG) parameters.

  4. Step 4

    Secondary assessments

    Secondary assessments include monitoring all-cause mortality and cardiovascular (CV) mortality.

    Changes from baseline in the distance walked during the 6-minute walk test (6MWT) and the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OS) are evaluated.

    CV-related hospitalizations and changes in transthyretin (TTR) levels are also assessed.

  5. Step 5

    Study duration

    The estimated end date for the study is May 9, 2028.

    The study began recruiting participants on October 1, 2021.

Who can join the trial?

4 criteria

  • Must have completed 30 months of the study treatment in a previous related study, including all required visits and procedures.
  • Must be able to understand and sign a written consent form before starting any study procedures.
  • Female participants who can have children and are sexually active with men must agree to use a **highly effective method of birth control** from the time they join the study until 30 days after their last dose of the study medication. If using birth control pills, they must also use another method of birth control. A **double-barrier method** (like using a condom with a diaphragm) is acceptable, even though it's not considered highly effective.
  • Male participants who are sexually active with women who can have children and have not had a vasectomy must agree to use a **double-barrier method** of birth control.

Who cannot join the trial?

4 criteria

  • Patients who do not have **symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM)** cannot participate. This is a condition where a protein called transthyretin builds up in the heart, causing symptoms.
  • Patients who are not within the specified age range cannot participate. The age range is typically defined in the study details.
  • Patients who do not meet the gender criteria cannot participate. Both males and females are eligible for this study.
  • Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups like children, pregnant women, or those unable to give consent.
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Investigated drugs

Acoramidis (AG10) is a medication being studied for its safety and tolerability in people with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM). This condition affects the heart, and the medication aims to help manage the symptoms associated with it. Participants in this study have previously completed a Phase 3 trial, and this extension study continues to monitor the effects of Acoramidis on their condition.

What is already known about the treatment

Acoramidis – Acoramidis is administered orally in tablet form and is currently being studied in clinical trials for its safety and tolerability in treating symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM). It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for acoramidis is to manage symptoms associated with ATTR-CM, a condition affecting the heart. At the molecular level, acoramidis works by stabilizing the transthyretin protein, preventing its misfolding and deposition in tissues. It is classified pharmacologically as a transthyretin stabilizer.

Investigated diseases

Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM) – This is a condition where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits are made of transthyretin, a protein that normally transports thyroid hormone and vitamin A in the blood. Over time, the buildup of amyloid can cause the heart walls to become stiff, leading to difficulties in the heart's ability to pump blood effectively. As the disease progresses, individuals may experience symptoms such as fatigue, shortness of breath, and swelling in the legs. The condition is considered rare and can significantly impact the quality of life.
Trial detailsLast updated 4 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515092-36-00Protocol codeAG10-304Estimated enrolment449 patientsSponsorEidos Therapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

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