Study of Vutrisiran for Patients with Transthyretin Amyloidosis and Heart Disease

3 1 1

What is this study about?

This clinical trial is focused on studying a condition known as Transthyretin Amyloidosis with Cardiomyopathy. This is a disease where abnormal proteins called amyloids build up in the heart, leading to heart problems. The study is testing a treatment called Vutrisiran, which is given as an injection under the skin. Vutrisiran is a type of medicine that belongs to a group known as nucleic acids, which are designed to target and reduce the production of these harmful proteins in the body.

The purpose of the study is to evaluate how effective Vutrisiran is compared to a placebo in reducing deaths from any cause and heart-related events, such as hospital visits due to heart problems. Participants in the study will receive either Vutrisiran or a placebo and will be monitored over a period of time to see how their condition changes. The study will also look at how Vutrisiran affects the ability to walk over a short distance, quality of life related to heart health, and overall health status.

Throughout the study, participants will undergo regular check-ups and assessments to track their health and any changes in their condition. The study aims to provide valuable information on whether Vutrisiran can be a safe and effective treatment option for people with Transthyretin Amyloidosis with Cardiomyopathy. This research could potentially lead to better management and outcomes for those affected by this challenging condition.

1 joining the study

Upon joining the study, the patient will be informed about the trial’s purpose, which is to evaluate the effectiveness and safety of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy.

The patient will be required to provide written informed consent, confirming their understanding and willingness to comply with the study requirements.

2 initial assessment

The patient will undergo an initial assessment to confirm eligibility, which includes a documented diagnosis of transthyretin amyloidosis with cardiomyopathy and a medical history of heart failure.

The patient must be clinically stable, with no cardiovascular-related hospitalizations within six weeks prior to randomization.

3 randomization

The patient will be randomly assigned to receive either vutrisiran or a placebo. This process is double-blind, meaning neither the patient nor the researchers will know which treatment the patient is receiving.

4 treatment administration

The patient will receive vutrisiran as a solution for injection, administered subcutaneously. The dosage is 25 mg, provided in a pre-filled syringe.

The frequency and duration of administration will be determined by the study protocol and communicated to the patient during the trial.

5 monitoring and follow-up

Throughout the trial, the patient will be monitored for any changes in health status, including cardiovascular events and overall mortality.

Regular assessments will include tests such as the 6-minute walk test and questionnaires to evaluate the impact on quality of life.

6 completion of the study

The study is estimated to conclude by November 21, 2025. Upon completion, the patient’s health outcomes will be evaluated to determine the efficacy and safety of vutrisiran.

Who Can Join the Study?

Age between 18 and 85 years.
Agree to sign a form to release medical records, if allowed by local rules, to collect information about health status and hospital visits during the study.
Have a confirmed diagnosis of ATTR amyloidosis with cardiomyopathy, which is a condition affecting the heart.
Have a history of heart failure with at least one previous hospital stay for heart failure, or show signs of heart failure that need treatment with a diuretic (a medicine that helps remove extra fluid from the body).
Meet one of these conditions: either have never used tafamidis (a medication) and do not plan to start it in the first year of the study, or are already using tafamidis as prescribed for heart issues.
Be in a stable health condition with no hospital visits related to heart problems in the last 6 weeks before starting the study.
Have a NT-proBNP level (a blood test that helps assess heart function) between 300 and 8500 ng/L, or between 600 and 8500 ng/L if you have a type of irregular heartbeat called atrial fibrillation.
Be able to walk at least 150 meters during a 6-minute walk test at the start of the study.
Have a Karnofsky performance status of 60% or higher, which means being able to carry out daily activities with some help.
Understand the study requirements and be willing and able to follow them, and provide written consent to participate.

Who Cannot Join the Study?

Patients who have a different type of amyloidosis other than Transthyretin Amyloidosis with Cardiomyopathy cannot participate. Amyloidosis is a condition where abnormal proteins build up in organs and tissues.
Patients who are not within the specified age range for the study cannot participate. The age range is determined by the study organizers.
Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
Patients who have other serious health 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 to participate.
Patients who have a history of drug or alcohol abuse that could affect the study results cannot participate.
Patients who have allergies to the study medication or its ingredients cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Oslo Universitetssykehus HF	Oslo	Norway
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Medical University Of Vienna	Vienna	Austria
Universitaetsmedizin Goettingen	Goettingen	Germany
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Oncopole Claudius Regaud	Toulouse	France
Katholieke Universiteit te Leuven	Leuven	Belgium
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland

Other Sites

Site Name	City	Country
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Lietuvos sveikatos mokslu universiteto ligonine Kauno klinikos	Kaunas	Lithuania
Synexus Czech s.r.o.	Prague	Czechia
Hospital Universitario Basurto	Bilbao	Spain
Unidade Local De Saude Do Alto Ave E.P.E.	Guimaraes	Portugal
O.L.Vrouw van Troost	Dendermonde	Belgium
KBC Zagreb	Zagreb	Croatia
Odense University Hospital	Odense	Denmark
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Semmelweis University	Budapest	Hungary
Norrlands University Hospital	Umea	Sweden
Synexus Polska Sp. z o.o.	Poznan	Poland
University Hospital Olomouc	Olomouc	Czechia
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
Stadt Wien Wiener Gesundheitsverbund	Vienna	Austria
Synexus Clinical Research GmbH	Frankfurt	Germany
Hospital Universitario Virgen De La Victoria	Malaga	Spain
Utyqavmxgxht Mnhlhdb Cryrprw Gfnpyktmc	Groningen	The Netherlands
Aqosyzvgkr Pwkknpuu Hjbmcjhr Dd Mzenayhly	Marseille	France
Avxsol Uxuiarelel Herquqjg	Aarhus	Denmark
Ucxxuhumgsqobsmapdjnp Mnffegbz Abq	Munster	Germany
Uwboqijrjitvxhqrefkcm Wdflfqaln Aje	Wuerzburg	Germany
Upugsvtamejhgqogxelff Ewyod Aex	Essen	Germany
Hpuwnboq Vpcs dxqomgso	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	21.11.2019
Belgium	Not recruiting	21.11.2019
Croatia	Not recruiting	21.11.2019
Czechia	Not recruiting	21.11.2019
Denmark	Not recruiting	21.11.2019
France	Not recruiting	21.11.2019
Germany	Not recruiting	21.11.2019
Hungary	Not recruiting	21.11.2019
Ireland	Not recruiting	21.11.2019
Lithuania	Not recruiting	21.11.2019
Norway	Not recruiting	21.11.2019
Poland	Not recruiting	21.11.2019
Portugal	Not recruiting	21.11.2019
Spain	Not recruiting	21.11.2019
Sweden	Not recruiting	21.11.2019
The Netherlands	Not recruiting	21.11.2019

Trial locations

Investigated drugs:

Vutrisiran

Vutrisiran is a medication being studied for its effectiveness in treating patients with a condition called transthyretin amyloidosis with cardiomyopathy. This condition affects the heart and can lead to serious heart problems. The trial aims to see if Vutrisiran can help reduce the risk of death and cardiovascular events in these patients.

Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy) – This is a rare disease where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits can cause the heart to become stiff, leading to difficulties in pumping blood effectively. Over time, this can result in symptoms such as fatigue, shortness of breath, and swelling in the legs and abdomen. The condition progresses as the amyloid deposits increase, further impairing heart function. Patients may experience worsening heart failure symptoms as the disease advances. The progression can vary among individuals, with some experiencing more rapid changes in heart function than others.

Trial ID:

2024-518318-25-00

Protocol code:

ALN-TTRSC02-003

Trial Phase:

Therapeutic confirmatory (Phase III)

Other Trials to Consider

#1 Clinical Trials Search in Europe

Study of Vutrisiran for Patients with Transthyretin Amyloidosis and Heart Disease

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?