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Study of Patisiran for Patients with Transthyretin Amyloidosis and Heart Disease

Fast replyRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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 trial?

10 criteria

  • 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 trial?

10 criteria

  • 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.
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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.

What is already known about the treatment

Patisiran – Patisiran is administered through intravenous infusion, typically in a clinical setting. It is currently being studied in a Phase 3 clinical trial for its effectiveness in treating transthyretin amyloidosis with cardiomyopathy, a condition where abnormal protein deposits affect heart function. The medication works by using RNA interference to reduce the production of transthyretin protein, which is responsible for the amyloid deposits. Patisiran is classified as a small interfering RNA (siRNA) therapeutic, targeting the genetic material involved in the disease process.

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-508364-29-00Protocol codeALN-TTR02-011Estimated enrolment278 patientsSponsorAlnylam Pharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).