Study on Long-Term Safety of Eplontersen for Adults with Transthyretin-Mediated Amyloid Cardiomyopathy

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What is this study about?

This clinical trial is focused on studying a condition known as Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM). This is a heart condition where abnormal proteins build up in the heart, affecting its function. The study will use a treatment called Eplontersen, which is also known by its code name ION 682884. Eplontersen is a type of medication called an antisense oligonucleotide, which is designed to target and reduce the production of specific proteins in the body. The purpose of this study is to evaluate the long-term safety of Eplontersen in patients with ATTR-CM.

Participants in the study will receive Eplontersen through a subcutaneous injection, which means it is injected under the skin. The study will also involve taking Concavit Capsules, which contain a combination of vitamins such as Vitamin A and Ascorbic Acid (Vitamin C). These capsules are taken orally, meaning they are swallowed. The study will last for a period of up to 36 months, during which participants will be monitored regularly to assess the safety and tolerability of the treatment. This includes checking various health indicators through blood tests and other clinical assessments.

The study aims to gather information on how the treatment affects the body over time, including any side effects or changes in health status. Participants will be asked to complete questionnaires about their quality of life and undergo tests to measure heart function and other health markers. The results of this study will help determine the long-term safety of Eplontersen for people with ATTR-CM.

1 joining the study

Upon joining the study, you will be informed about the purpose and procedures involved. This study aims to assess the long-term safety of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

You will be required to have completed a previous study or have a diagnosis of ATTR-CM and satisfactory participation in a related study, as determined by the investigator.

2 medication administration

You will receive eplontersen as a solution for injection. This will be administered through a subcutaneous injection, which means it is injected under the skin.

The frequency and dosage of the medication will be determined by the study protocol and the investigator overseeing your participation.

3 vitamin A supplementation

As part of the study, you will be required to adhere to a vitamin A supplementation regimen. This is to ensure your safety and the effectiveness of the treatment.

4 regular health assessments

Throughout the study, you will undergo regular health assessments. These will include laboratory tests, physical examinations, and electrocardiograms (ECGs) to monitor your health and the effects of the medication.

The assessments will focus on various health indicators such as platelet count, kidney function, liver function, thyroid function, and coagulation tests.

5 monitoring side effects

You will be monitored for any side effects or adverse reactions to the medication. This includes tracking any unexpected symptoms or changes in your health.

The study will also assess the presence of any anti-drug antibodies, which are immune responses that can occur with certain medications.

6 biomarker and clinical tests

The study will involve analysis of various biomarkers and clinical tests over time. Biomarkers are substances in your body that can indicate how well your body is responding to the treatment.

Tests will include measurements of transthyretin serum levels, walking tests, echocardiograms, and quality of life questionnaires.

7 completion of the study

The study is expected to continue until August 2029. Your participation will involve regular visits and assessments as outlined in the study protocol.

Upon completion, you will have a final assessment to evaluate your overall health and the long-term effects of the treatment.

Who Can Join the Study?

Must have completed the Treatment Period and the End of Treatment Visit of the previous study (ION-682884-CS2) OR have a diagnosis of Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) and satisfactory participation in the ISIS 420915CS101 study, as judged by the study doctor and sponsor.
The study doctor must be willing to treat the participant with open label ION-682884 (eplontersen).
Must be willing to take vitamin A supplements as required by the study protocol.
Both men and women can participate.
Participants must be within the specified age range for the study.

Who Cannot Join the Study?

Patients who have a different heart condition other than Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) cannot participate.
Patients who are not within the specified age range for the study cannot participate.
Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
Patients who have any other serious medical conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have participated in another clinical trial recently may not be eligible.
Patients who have allergies to the study medication or its ingredients cannot participate.
Patients who have a history of substance abuse or alcohol abuse cannot participate.
Patients who have any condition that the study doctors believe would make it unsafe for them to participate cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medical University Of Graz	Graz	Austria
Medical University Of Vienna	Vienna	Austria
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Medizinische Universitaet Innsbruck	Innsbruck	Austria
Azienda Ospedaliero Universitaria Careggi	Florence	Italy

Other Sites

Site Name	City	Country
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Alexandra Hospital	Athens	Greece
Az St-Jan Brugge-Oostende A.V.	Brugge	Belgium
University Clinical Hospital Virgen De La Arrixaca	Murcia	Spain
Fakultni Nemocnice U Sv Anny V Brne	Brno-Stred	Czechia
Azienda Ospedaliero-Universitaria Sant Andre	Rome	Italy
Azienda Ospedaliera di Padova	Padua	Italy
Centre Hospitalier Universitaire De Toulouse	Toulouse	France
Saarland University Hospital	Homburg	Germany
Universita’ Politecnica Delle Marche	Ancona	Italy
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Universitaetsklinikum Wuerzburg AöR	Wuerzburg	Germany
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
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Unidade Local De Saude Do Alto Ave E.P.E.	Guimaraes	Portugal
Centre Hospitalier Universitaire De Nantes	Saint-Herblain	France
Westfaelische Wilhelms Universitaet Muenster	Munster	Germany
Västra Götalandregionen	Gothenburg	Sweden
Fotpouuamz Ttraczx Gopvhgin Mebpbypybq	Pisa	Italy
Rfwnpz Vpwpyfmosoxk	Skellefteå	Sweden
Uyndtjfqwn Hiulvcmr Cowqrbk	Cologne	Germany
Agbujqsjvs Pzdinxlv Hvsdvbxw Da Muzdyztry	Marseille	France
Imhroelvc Fpn Cbkrsfez Acw Eoyestvcxljn Mqiqpvxn	Prague	Czechia
Hozgocdu Vfkp dpbcrgqj	Barcelona	Spain
Hlnhcyj Hhsxb Meljhm &pxomgo 1 rfh Ghfqbnw Eadlst	Creteil	France
Klystftfy Srcomnm Skordplcjpyauiz im Jkjm Pfurw Iv	Cracow	Poland

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	08.06.2023
Belgium	Not recruiting	08.06.2023
Czechia	Not recruiting	08.06.2023
Denmark	Not recruiting	08.06.2023
France	Not recruiting	08.06.2023
Germany	Not recruiting	08.06.2023
Greece	Not recruiting	08.06.2023
Italy	Not recruiting	08.06.2023
Poland	Not recruiting	08.06.2023
Portugal	Not recruiting	08.06.2023
Spain	Not recruiting	08.06.2023
Sweden	Not recruiting	08.06.2023

Trial locations

Investigated drugs:

Eplontersen is a medication being studied for its long-term safety in patients with a condition called transthyretin-mediated amyloid cardiomyopathy, or ATTR-CM. This condition affects the heart and can lead to heart failure. Eplontersen is designed to help manage this condition by targeting and reducing the production of a specific protein that contributes to the disease. The goal of using Eplontersen in this trial is to see how well patients tolerate the medication over an extended period and to ensure it is safe for long-term use.

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) – This disease is characterized by the buildup of abnormal protein deposits called amyloid in the heart tissue. These deposits are formed from transthyretin, a protein that normally transports thyroid hormones and vitamin A in the blood. Over time, the accumulation of amyloid disrupts the normal structure and function of the heart, leading to thickening and stiffening of the heart walls. This results in impaired heart function, which can cause symptoms such as fatigue, shortness of breath, and swelling in the legs. The disease progresses as the amyloid deposits continue to accumulate, further compromising heart function. It is a rare condition that can be hereditary or occur without a family history.

Trial ID:

2022-502415-11-00

Protocol code:

ION-682884-CS12

NCT ID:

NCT05667493

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-Term Safety of Eplontersen for Adults with Transthyretin-Mediated Amyloid Cardiomyopathy

What is this study about?

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Who Cannot Join the Study?