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Study on Acoramidis for Preventing Transthyretin Amyloidosis in Young People with a Genetic Risk

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What is this trial about?

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

The study focuses on a condition called Transthyretin Amyloidosis, which is a rare disease that can affect the heart and nerves. The trial is investigating a treatment called Acoramidis (AG10), which is taken as a tablet. This treatment is being compared to a placebo to see if it can prevent the disease in people who carry a specific genetic variant but do not yet show symptoms.

The purpose of the study is to determine if Acoramidis can effectively prevent the development of Transthyretin Amyloidosis in individuals who are at risk but currently asymptomatic. Participants will be randomly assigned to receive either the treatment or a placebo. The study will monitor participants over time to see if they develop symptoms of the disease.

The trial is designed to be a long-term study, with participants being followed for several years. The main focus will be on the time it takes for the disease to develop in those taking the treatment compared to those taking the placebo. The study will also look at other health outcomes and any side effects that may occur during the trial period.

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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, genetic status, and other criteria.

    Participants must have a specific genetic variant related to transthyretin amyloidosis and be within the age range of 18 to 75 years.

  2. Step 2

    Randomization

    Participants are randomly assigned to receive either the acoramidis tablet or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

  3. Step 3

    Medication administration

    Participants take the assigned tablet orally. The dosage and frequency are determined by the study protocol and are consistent throughout the trial.

    The duration of medication administration continues until the study's estimated end date in November 2031, unless otherwise specified by the study protocol.

  4. Step 4

    Regular monitoring

    Participants undergo regular health assessments to monitor for any signs of transthyretin amyloidosis or other health changes.

    These assessments include physical examinations, vital sign checks, and laboratory tests to ensure participant safety and gather data on the medication's effects.

  5. Step 5

    End of study evaluation

    At the conclusion of the study, a final evaluation is conducted to assess the participant's health and any developments related to transthyretin amyloidosis.

    The primary focus is on the time to development of any symptoms or conditions related to the disease.

Who can join the trial?

5 criteria

  • Participants must be willing and able to give a signed informed consent for study procedures. This means they agree to take part in the study after understanding what it involves.
  • Participants must be between 18 and 75 years old when signing the informed consent.
  • Participants must have a specific change in their DNA, called a **TTR gene variant**, which is known to cause the condition. This must be confirmed by a special test before joining the study.
  • Participants' age should be no more than 10 years younger than the age when their family members typically show symptoms of the condition, known as **PADO**. For example, if family members usually show symptoms at 50 years old, participants should be at least 40 years old.
  • Participants must agree to use highly effective birth control methods during the study and for 30 days after the last dose of the study drug. This applies to both men and women who can have children.

Who cannot join the trial?

2 criteria

  • Individuals who already show signs of **Transthyretin Amyloidosis** cannot participate. This is a condition where a protein called transthyretin builds up in the body and can cause health problems.
  • Participants must not have any clinical evidence of the disease at the start of the study. This means they should not have any symptoms or medical findings that suggest they have the condition.
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Investigated drugs

Acoramidis is a medication being studied for its ability to prevent a condition called transthyretin amyloidosis (ATTR) in people who carry a specific genetic variant. This condition can lead to problems with the heart or nerves. The trial is focused on individuals who have the genetic variant but do not yet show any symptoms of the disease. The goal is to see if acoramidis can stop or delay the onset of symptoms related to this condition.

What is already known about the treatment

Acoramidis – Acoramidis is administered orally and is currently being studied in clinical trials for its potential to prevent transthyretin amyloidosis, a condition where abnormal protein deposits affect organs and tissues. The medication is in the phase 3 stage of clinical trials, focusing on its efficacy in preventing the onset of amyloidosis in individuals who carry a genetic variant but do not yet show symptoms. Acoramidis works by stabilizing the transthyretin protein, preventing it from misfolding and forming harmful deposits. It is classified as a transthyretin stabilizer, aiming to maintain normal protein function and prevent disease progression.

Investigated diseases

Transthyretin Amyloidosis – This is a rare disease where abnormal protein deposits, known as amyloid, accumulate in the body's tissues and organs. It is caused by mutations in the transthyretin (TTR) gene, leading to the production of unstable proteins that misfold and form amyloid fibrils. These deposits can affect various organs, most commonly the heart and nerves, leading to conditions such as cardiomyopathy and peripheral neuropathy. The disease progresses as more amyloid builds up, causing organ dysfunction and related symptoms. It can manifest in different forms, including hereditary and wild-type, with varying symptoms depending on the organs involved. Early stages may be asymptomatic, but as the disease advances, it can lead to significant health issues.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513547-82-00Protocol codeAG10-501Estimated enrolment637 patientsSponsorEidos Therapeutics Inc.

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