Study of Patisiran for 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 called amyloids build up in the heart, affecting its function. The study is evaluating a treatment called Patisiran, which is administered as a solution for infusion. Patisiran is designed to target and reduce the production of transthyretin, a protein that can misfold and form amyloid deposits in the heart.

The purpose of the study is to assess how effective and safe Patisiran is compared to a placebo in improving the ability to walk, as measured by a test called the 6-minute walk test. Participants in the study will receive either Patisiran or a placebo through an intravenous infusion, which means the medication is given directly into a vein. The study will last for about 12 months, during which participants will have regular check-ups to monitor their health and the effects of the treatment.

Throughout the study, researchers will also look at other health outcomes, such as changes in symptoms and overall quality of life, using tools like the Kansas City Cardiomyopathy Questionnaire. The study aims to provide valuable information on whether Patisiran can help improve the lives of people with ATTR Amyloidosis with Cardiomyopathy by reducing symptoms and improving heart function.

1 joining the study

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

2 treatment administration

The patient will receive patisiran as an intravenous infusion. The medication is provided as a 2 mg/mL concentrate for solution for infusion, known as Onpattro.

The infusion will be administered regularly, but the specific frequency and duration are determined by the study protocol and the patient’s condition.

3 monitoring and assessments

Throughout the study, the patient’s health and response to the treatment will be closely monitored. This includes regular assessments of functional capacity using the 6-minute walk test (6-MWT).

Additional evaluations may include questionnaires like the Kansas City Cardiomyopathy Questionnaire to assess the impact on quality of life.

4 completion of the study

The study is designed to last for a period of 12 months. At the end of this period, the patient’s progress will be evaluated based on changes from the baseline in the 6-MWT and other health indicators.

The study aims to determine the efficacy and safety of patisiran in treating transthyretin amyloidosis with cardiomyopathy.

Who Can Join the Study?

  • Must be between 18 and 85 years old.
  • Must have a documented diagnosis of ATTR amyloidosis with cardiomyopathy. This means a specific type of heart condition caused by protein deposits.
  • Must have a history of heart failure (HF), which is when the heart doesn’t pump blood as well as it should. This includes at least one past hospital stay for heart failure or current signs of heart failure that need treatment with a diuretic (a medicine that helps remove excess fluid from the body).
  • Must meet one of the following conditions regarding tafamidis (a medication used for this condition):
    • Never taken tafamidis or taken it for 30 days or less and not in the last 6 months.
    • Currently taking tafamidis for at least 6 months and the condition is getting worse.
  • Must be clinically stable, meaning no hospital visits related to heart problems in the last 6 weeks.
  • Must be able to walk at least 150 meters in a 6-minute walk test, which measures how far you can walk in 6 minutes.
  • Must have specific levels of NT-proBNP, a blood test that helps assess heart function. Levels should be between 300 and 8500 ng/L, or between 600 and 8500 ng/L if you have a type of irregular heartbeat called atrial fibrillation.
  • Must be able to understand and agree to follow the study requirements and provide written consent to participate.

Who Cannot Join the Study?

  • Patients who have other serious health conditions that could interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a heart transplant or are on a waiting list for one.
  • Patients who have had a recent heart attack or stroke.
  • Patients who have severe kidney disease.
  • Patients who have severe liver disease.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who are unable to walk or perform the 6-minute walk test.
  • Patients who have an allergy to the study medication or its ingredients.

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
University Hospital Maastricht Maastricht The Netherlands
Oncopole Claudius Regaud Toulouse France

Other Sites

Site Name City Country Status
Centre Hospitalier Regional De La Citadelle Liege Belgium
Algemeen Ziekenhuis Delta Roeselare Belgium
Synexus Czech s.r.o. Prague Czechia
Odense University Hospital Odense Denmark
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Karolinska University Hospital Solna Sweden
Jessa Ziekenhuis Hasselt Belgium
Region Midtjylland Aarhus Denmark
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Centre Hospitalier Universitaire De Rennes Rennes France
Rigshospitalet Copenhagen Denmark
A.O.U. Policlinico G. Martino Di Messina Messina Italy
Hopital Beaujon Clichy France
Ihqrsciui Fch Cpsmdnbv Aad Enjsthnvsmbm Maskrluo Prague Czechia
Ultcqfdcwdfe Mpcyxpo Cqpjltl Gupcrvpcg Groningen The Netherlands
Ozgztwzgzugwpbdycgnaffdsfi Aalst Belgium
Acqdjgr Ulrar Suatqzmko Lkbyst Dy Bdvvyji Bologna Italy

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
16.01.2020
Czechia Czechia
Not recruiting
16.01.2020
Denmark Denmark
Not recruiting
16.01.2020
France France
Not recruiting
16.01.2020
Italy Italy
Not recruiting
16.01.2020
Portugal Portugal
Not recruiting
16.01.2020
Sweden Sweden
Not recruiting
16.01.2020
The Netherlands The Netherlands
Not recruiting
16.01.2020

Trial locations

Investigated drugs:

Patisiran 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 heart failure. The study aims to see how well patisiran can improve the ability of patients to perform physical activities, measured by a test where patients walk for six minutes.

Investigated diseases:

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. The condition progresses as more amyloid builds up, further impairing heart function. It primarily affects older adults and can be hereditary or occur without a family history. The disease’s progression can vary, with some individuals experiencing a gradual worsening of symptoms.

Trial ID:
2023-508364-29-00
Protocol code:
ALN-TTR02-011
Trial Phase:
Therapeutic confirmatory (Phase III)

Other Trials to Consider

  • A Study of Acoramidis in Adult Patients with Transthyretin Amyloidosis with Cardiomyopathy Who Previously Received Tafamidis

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    Investigated drugs:
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  • A study of coramitug compared to placebo for people with heart failure caused by transthyretin amyloid cardiomyopathy

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