Study to Evaluate the Safety and Effectiveness of Vutrisiran in Patients with Transthyretin Amyloidosis and Heart Disease

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What is this study about?

This clinical trial is focused on studying a condition known as Transthyretin Amyloidosis with Cardiomyopathy, often referred to as ATTR Amyloidosis with Cardiomyopathy. This is a disease where abnormal proteins build up in the heart, affecting its function. The study will use a treatment called Vutrisiran, which is a solution for injection. Vutrisiran is also known by its code name ALN-TTRSC02. The purpose of the study is to gather information on the safety of Vutrisiran in patients who have this condition.

Participants in the study will receive Vutrisiran through an injection under the skin. The study will observe patients who are continuing their treatment with Vutrisiran or those who are switching from another medication called Patisiran. The study will last for a period of time, during which the safety of the treatment will be closely monitored. Patients will be asked to take vitamin A supplements as part of the study protocol.

The study will track how often any side effects occur and will also look at changes in heart-related health measures. These include changes in a questionnaire score that assesses heart failure symptoms, changes in heart function class, and levels of certain proteins in the blood that indicate heart stress. The study will also monitor overall survival and any heart-related hospital visits. The goal is to better understand how Vutrisiran affects patients with ATTR Amyloidosis with Cardiomyopathy over an extended period.

1 joining the study

Participation begins after completing the required parent studies or the 24-month period in the vutrisiran parent study.

Written informed consent is necessary to proceed.

2 medication administration

Receive Amvuttra (vutrisiran) 25 mg solution for injection.

The medication is administered through a subcutaneous injection, which means it is injected under the skin.

3 supplementation

Adhere to vitamin A supplementation as outlined in the study protocol.

4 monitoring and assessments

Regular monitoring for any adverse events is conducted to ensure safety.

Assessments include changes from baseline in heart-related health scores and biomarkers.

Monitoring of overall health outcomes, including mortality and cardiovascular events, is performed.

5 study duration

The study is expected to continue until June 1, 2027.

Participation involves ongoing assessments and adherence to the study protocol throughout this period.

Who Can Join the Study?

  • Patients must have ATTR amyloidosis with cardiomyopathy. This is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients should be participating in specific studies related to the condition, such as the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014, or have completed a 24-month period in the vutrisiran parent study ALN-TTRSC02-003.
  • Patients must be willing to take vitamin A supplements as part of the study requirements.
  • The patient or their legal guardian must be able to understand the study requirements and agree to participate by signing a written consent form.
  • Both male and female patients are eligible to participate.

Who Cannot Join the Study?

  • Patients who have a different type of amyloidosis other than Transthyretin Amyloidosis with Cardiomyopathy. This is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients who are not in the specified age range for the study. The study is open to certain age groups only.
  • Patients who are not able to continue using the study medication, vutrisiran, or who have not switched from another medication called patisiran.
  • Patients who are not able to provide informed consent, which means they cannot understand the study and agree to participate.
  • Patients who are part of a vulnerable population, meaning they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Careggi Florence Italy
Oslo Universitetssykehus HF Oslo Norway
Bellvitge University Hospital L'hospitalet De Llobregat Spain
Medical University Of Vienna Vienna Austria
Katholieke Universiteit te Leuven Leuven Belgium
University Hospital Maastricht Maastricht The Netherlands
Oncopole Claudius Regaud Toulouse France
Unidade Local De Saúde De Santa Maria, E.P.E. Lisbon Portugal
Université Libre de Bruxelles – Hôpital Erasme Brussels Belgium

Other Sites

Site Name City Country Status
Region Vaesterbotten Umea Sweden
Universitair Ziekenhuis Gent Gent Belgium
Centre Hospitalier Regional De La Citadelle Liege Belgium
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Algemeen Ziekenhuis Delta Roeselare Belgium
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
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Karolinska University Hospital Solna Sweden
Jessa Ziekenhuis Hasselt Belgium
Centre Hospitalier Universitaire De Rennes Rennes France
Rigshospitalet Copenhagen Denmark
Odense University Hospital Odense Denmark
Region Midtjylland Aarhus Denmark
Vseobecna Fakultni Nemocnice V Praze Prague 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
Hospital Universitario Virgen De La Victoria Malaga Spain
Itrzjvkix Ftn Cmoskprk Agm Eizprvysfuux Myyhgzos Prague Czechia
Usczjuqmramw Moltvtj Cchdxot Gztopdwox Groningen The Netherlands
Olgevtexkqyimpxyxhlbkxspzc Aalst Belgium
Apiqbpiexy Pyvpzdgq Hgvxpdsi Dh Mhcgtccwv Marseille France
Aaljarl Uggha Slxpbsqia Lpvkxo Dx Bsbszbk Bologna Italy
Hjdsotcj Vjop dhgoorpz Barcelona Spain

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
29.01.2025
Belgium Belgium
Not recruiting
29.01.2025
Czechia Czechia
Not recruiting
29.01.2025
Denmark Denmark
Not recruiting
29.01.2025
France France
Not recruiting
29.01.2025
Italy Italy
Not recruiting
29.01.2025
Norway Norway
Not recruiting
29.01.2025
Portugal Portugal
Not recruiting
29.01.2025
Spain Spain
Not recruiting
29.01.2025
Sweden Sweden
Not recruiting
29.01.2025
The Netherlands The Netherlands
Not recruiting
29.01.2025

Trial locations

Investigated drugs:

Vutrisiran is a medication being studied for its safety and effectiveness in patients with a condition called transthyretin amyloidosis with cardiomyopathy. This condition affects the heart and is caused by abnormal proteins. Vutrisiran is designed to help reduce the production of these abnormal proteins, potentially improving heart function and symptoms in affected patients.

Patisiran is another medication used in the study. It is also aimed at treating transthyretin amyloidosis by reducing the levels of abnormal proteins in the body. Some patients in the trial may have switched from using patisiran to vutrisiran to see if there are differences in safety or effectiveness between the two treatments.

Investigated diseases:

Transthyretin 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. The condition progresses as the amyloid deposits increase, further impairing heart function. It is often associated with genetic mutations that affect the transthyretin protein. The disease primarily affects older adults and can significantly impact quality of life.

Trial ID:
2024-518343-38-00
Protocol code:
ALN-TTRSC02-007
Trial Phase:
Therapeutic confirmatory (Phase III)

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