#1 Clinical Trials Search in Europe

Acoramidis Hydrochloride

Acoramidis Hydrochloride, also known as AG10, is a drug currently being studied in clinical trials for the treatment of symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM). This article explores the ongoing research into this promising medication, its potential benefits, and what patients with ATTR-CM can expect from participating in these trials.

Table of Contents

What is Acoramidis?

Acoramidis, also known as AG10, is a new medication being studied for the treatment of symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM). It is currently in the clinical trial phase and is not yet approved for general use. Acoramidis is taken as a tablet by mouth and is being developed by Eidos Therapeutics, Inc.[1]

What is Transthyretin Amyloid Cardiomyopathy (ATTR-CM)?

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a serious heart condition. In this disease, a protein called transthyretin (TTR) becomes unstable and forms clumps (amyloid) in the heart. These clumps make it harder for the heart to work properly, leading to symptoms like shortness of breath, fatigue, and swelling in the legs.[1]

How Does Acoramidis Work?

Acoramidis is designed to work by stabilizing the transthyretin (TTR) protein. By keeping TTR stable, it aims to prevent the formation of amyloid clumps in the heart. This could potentially slow down or stop the progression of ATTR-CM.[1]

Current Clinical Trial

A clinical trial is currently underway to study the long-term safety and effectiveness of Acoramidis. This trial is called an “open-label extension study,” which means that all participants receive the actual medication (not a placebo). The study will last for up to 60 months (5 years) and includes patients who completed a previous 30-month study of Acoramidis.[1]

Potential Benefits of Acoramidis

The researchers are looking at several potential benefits of Acoramidis, including:

  • Improved survival rates (both overall and specifically related to heart problems)
  • Better ability to walk (measured by a 6-minute walk test)
  • Improved quality of life (measured by a special questionnaire for heart failure patients)
  • Fewer hospitalizations due to heart problems
  • Stabilization of the TTR protein in the body

It’s important to note that these are potential benefits being studied, and the actual effects of the medication are still being determined.[1]

Safety Considerations

The main goal of this study is to assess the safety of Acoramidis when used for a long time. The researchers will be closely monitoring:

  • Any serious side effects
  • Side effects that cause patients to stop taking the medication
  • Changes in physical exams, vital signs, heart tests (ECGs), and laboratory tests

This careful monitoring helps ensure the safety of participants and provides important information about how the medication affects the body over time.[1]

Who Can Participate in the Study?

The study is designed for people who:

  • Have completed a previous 30-month study of Acoramidis
  • Have symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM)
  • Are able to understand and sign an informed consent form

There are also several factors that would prevent someone from participating, such as:

  • Having unstable heart function
  • Recent heart or liver transplant
  • Having a different type of amyloidosis (AL amyloidosis)
  • Severe kidney problems
  • Known allergy to Acoramidis

If you think you might be eligible for this or similar studies, it’s important to discuss this with your doctor. They can provide more information and help determine if participating in a clinical trial is right for you.[1]

Aspect Details
Drug Name Acoramidis Hydrochloride (AG10)
Condition Studied Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Study Type Open-Label Extension and Safety Monitoring Study
Main Objective Assess safety and tolerability of Acoramidis in ATTR-CM patients
Secondary Objectives Evaluate effects on mortality, physical function, quality of life, and hospitalization
Treatment Duration Up to 60 months
Key Eligibility Completed 30 months of previous AG10-301 study
Primary Endpoints Safety parameters including adverse events and laboratory findings
Secondary Endpoints Mortality, 6-minute walk test, quality of life, hospitalization, TTR levels

FAQ

  • What is transthyretin amyloid cardiomyopathy (ATTR-CM)? ATTR-CM is a heart condition where abnormal proteins called amyloids build up in the heart, affecting its function. This can lead to heart failure and other serious complications.
  • What is the main goal of the Acoramidis clinical trial? The main goal is to assess the safety and tolerability of Acoramidis in patients with symptomatic ATTR-CM who have completed a previous 30-month study.
  • How long does the Acoramidis clinical trial last? The treatment period lasts up to approximately 60 months (5 years), with a follow-up visit 30 days after the last dose.
  • What are some of the key things researchers will be looking at during the trial? Researchers will be monitoring safety, all-cause mortality, cardiovascular mortality, changes in the 6-minute walk test, quality of life, and the frequency of cardiovascular-related hospitalizations.
  • Who can participate in this clinical trial? Participants must have completed 30 months of a previous study (AG10-301), be able to provide informed consent, and meet specific criteria regarding their health status and willingness to use contraception if applicable.

Ongoing Clinical Trials on Acoramidis Hydrochloride

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

    Recruiting

    3 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Denmark France Germany Greece +6

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

    Not yet recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Denmark France Germany Greece Ireland +5

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

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia Denmark Greece Ireland Italy +3

Glossary

  • Transthyretin Amyloid Cardiomyopathy (ATTR-CM): A heart disease caused by the buildup of abnormal protein deposits (amyloids) in the heart, affecting its function and potentially leading to heart failure.
  • Acoramidis (AG10): The drug being studied in this clinical trial for the treatment of ATTR-CM.
  • Open-Label Extension Study: A type of clinical trial where all participants receive the study drug and both researchers and participants know what treatment is being given.
  • 6-Minute Walk Test (6MWT): A test that measures the distance a person can walk in 6 minutes, used to assess physical function and endurance.
  • Kansas City Cardiomyopathy Questionnaire (KCCQ): A survey used to measure health-related quality of life in patients with heart failure.
  • Cardiovascular (CV): Relating to the heart and blood vessels.
  • Pharmacodynamic (PD): The study of how a drug affects the body.
  • TTR: Transthyretin, a protein in the blood that can form abnormal deposits in ATTR-CM.
  • Adverse Event (AE): Any unfavorable medical occurrence in a patient during a clinical study, whether or not it is related to the study treatment.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization, or causes significant disability.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-of-acoramidis-in-patients-with-transthyretin-amyloid-cardiomyopathy-who-completed-a-previous-trial/