Study on the Effectiveness and Safety of Eplontersen for Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

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What is this study about?

This clinical trial is focused on studying a condition called Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM). ATTR-CM is a heart disease where abnormal proteins build up in the heart, affecting its function. The study will test a treatment called ION-682884, also known as Eplontersen, which is a type of medication called an antisense oligonucleotide. This medication is designed to target and reduce the production of the abnormal protein causing the disease. Participants in the study will receive either the ION-682884 treatment or a placebo, which is an inactive substance that looks like the treatment but does not contain the active medication.

The purpose of the study is to evaluate the effect of ION-682884 on heart-related health outcomes in patients with ATTR-CM. Participants will receive regular injections under the skin and will be monitored over a period of time to assess the treatment’s impact on their heart health. The study will compare the number of heart-related events, such as heart failure or heart attacks, between those receiving the treatment and those receiving the placebo. The study aims to determine if ION-682884 can improve the health and quality of life for people with ATTR-CM.

Throughout the study, participants will continue to receive their usual care for ATTR-CM. The study will involve regular visits to the clinic for health assessments and to receive the injections. The overall goal is to see if ION-682884 can reduce the risk of serious heart problems and improve the well-being of patients with this condition. Participants’ health and safety will be closely monitored by the study team throughout the trial.

1 joining the study

Upon joining the study, participation is confirmed through a process that ensures eligibility based on specific criteria related to health and medical history.

Participants must meet certain health conditions, such as having a confirmed diagnosis of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) and a history of heart failure.

2 randomization

Participants are randomly assigned to one of two groups: one receiving the active medication, ION-682884, and the other receiving a placebo.

The study is double-blind, meaning neither the participants nor the researchers know who is receiving the active medication or the placebo.

3 medication administration

The active medication, ION-682884, or the placebo is administered as a subcutaneous injection.

The placebo consists of a sterile solution of sodium chloride and riboflavin in water for injection.

4 treatment duration

The treatment continues over a period that extends until the estimated end date of the study, which is November 15, 2025.

Participants are monitored regularly to assess the effects of the treatment on cardiovascular health and overall well-being.

5 evaluation of outcomes

The primary goal is to evaluate the effect of ION-682884 on cardiovascular outcomes, including death and clinical events related to heart health.

Secondary outcomes include changes in walking distance and quality of life scores, assessed at specific intervals during the study.

6 completion of the study

At the end of the study, data from all participants are analyzed to determine the effectiveness and safety of ION-682884 compared to the placebo.

The results contribute to understanding the potential benefits of the medication for patients with ATTR-CM.

Who Can Join the Study?

  • Females must not be pregnant or breastfeeding. They should be either surgically sterile, post-menopausal, or not engaging in sexual activity. If they are sexually active and can have children, they must agree to use one highly effective birth control method.
  • If taking medication for heart failure, the dosage should be stable for at least two weeks before joining the study.
  • Participants must agree to take vitamin A supplements as outlined in the study plan.
  • Males must be surgically sterile, not engaging in sexual activity, or if sexually active with a woman who can have children, they or their partner must use a highly effective birth control method.
  • Participants must agree to genetic testing for changes in the transthyretin (TTR) gene if it hasn’t been done before.
  • Amyloid deposits must be confirmed in heart or other tissues using special staining or imaging tests, with specific results, and no abnormal light chains ratio.
  • The thickness of the heart’s interventricular septum must be more than 12 mm on an echocardiogram during screening.
  • Participants must have a history of heart failure due to hereditary or wild-type ATTR-CM, with either a previous hospital stay for heart failure or related issues, or symptoms that require treatment with diuretics (except certain types) for stabilization.
  • The level of a specific heart-related protein, N-terminal prohormone of brain natriuretic peptide (NT-proBNP), must be at least 600 pg/mL, or 1200 pg/mL if the participant has atrial fibrillation, as measured by a central lab.
  • Participants must be classified as New York Heart Association (NYHA) class I-III, which indicates the severity of heart failure symptoms.
  • Participants must be able to walk at least 100 meters in a 6-minute walk test (6MWT).

Who Cannot Join the Study?

  • Patients who have a different heart condition that is not related to Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM).
  • Patients who are not receiving the standard care available for their condition.
  • Patients who are not within the specified age range for the study.
  • Patients who are not able to follow the study procedures or attend the required visits.
  • Patients who have other serious health issues that might interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients who have allergies or reactions to the study medication or similar drugs.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Universitaet Innsbruck Innsbruck Austria
Azienda Ospedaliero Universitaria Careggi Florence Italy
Medical University Of Vienna Vienna Austria
Universitaetsklinikum Heidelberg AöR Heidelberg Germany
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Medical University Of Graz Graz Austria
Centro Hospitalar Universitario Sao Joao E.P.E. Porto Portugal
Oncopole Claudius Regaud Toulouse France
Comite Entreprise Paul Papin Angers France

Other Sites

Site Name City Country Status
Centre Hospitalier Departemental Vendee La Roche sur Yon France
Azienda Ospedaliero-Universitaria Sant Andre Rome Italy
Fondazione Toscana Gabriele Monasterio Pisa Italy
Region Vaesterbotten Umea Sweden
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy Warsaw Poland
Unidade Local De Saude Do Alto Ave E.P.E. Guimaraes Portugal
Azienda Ospedaliera di Padova Padua Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Alexandra Hospital Athens Greece
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Saarland University Hospital Homburg Germany
University Clinical Hospital Virgen De La Arrixaca Murcia Spain
Universita’ Politecnica Delle Marche Ancona Italy
Universidade De Santiago De Compostela Santiago De Compostela Spain
Fakultni Nemocnice U Sv Anny V Brne Brno-Stred Czechia
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Ziekenhuis Oost Limburg Genk Belgium
Rigshospitalet Copenhagen Denmark
Centro Hospitalar Universitario De Santo Antonio E.P.E. Porto Portugal
Hopital Beaujon Clichy France
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Unidade Local De Saude De Sao Jose E.P.E. Lisbon Portugal
Iuebmoroa Fsm Cyexalau Ajb Ervurtvfxbvk Mrosdizc Prague Czechia
Ufquyktxst Hkenwulr Ciulopw Cologne Germany
Akgxcikarw Polctevm Huhukqqo Dh Mmzdckoqt Marseille France
Uallrslnsudzbgbqjpgdz Mystikae Adw Munster Germany
Ufaybipqeamithhsvqgto Wjtgelfdj Acs Wuerzburg Germany
Hcrkigki Vtvk dlyowrzf Barcelona Spain
Kyncwbgxn Sfrbwdv Slpvzdqqzpotlpi ir Jpcs Pdgri Iw Cracow Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
13.03.2020
Belgium Belgium
Not recruiting
13.03.2020
Czechia Czechia
Not recruiting
13.03.2020
Denmark Denmark
Not recruiting
13.03.2020
France France
Not recruiting
13.03.2020
Germany Germany
Not recruiting
13.03.2020
Greece Greece
Not recruiting
13.03.2020
Italy Italy
Not recruiting
13.03.2020
Poland Poland
Not recruiting
13.03.2020
Portugal Portugal
Not recruiting
13.03.2020
Spain Spain
Not recruiting
13.03.2020
Sweden Sweden
Not recruiting
13.03.2020

Trial locations

Investigated drugs:

ION-682884 is a medication being studied for its potential to help patients with a condition called transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This condition affects the heart and can lead to heart failure. The medication is being tested to see if it can reduce the risk of cardiovascular death and other heart-related problems in patients who are already receiving standard treatments for their condition.

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) – This is a condition where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits are made up 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, patients may experience symptoms such as fatigue, shortness of breath, and swelling in the legs. The condition can also lead to complications like heart failure and arrhythmias. ATTR-CM is considered a rare disease, affecting a small percentage of the population.

Trial ID:
2024-514434-20-00
Protocol code:
ION-682884-CS2
NCT ID:
NCT04136171
Trial Phase:
Therapeutic confirmatory (Phase III)

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