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Study on the Effectiveness and Safety of ALXN2220 for Adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

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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 Amyloid Cardiomyopathy (ATTR-CM). ATTR-CM is a heart disease caused by the buildup of abnormal proteins called amyloids in the heart tissue, which can lead to heart failure. The study is testing a new treatment called ALXN2220, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins made in a lab that can bind to specific substances in the body, in this case, the amyloid proteins, to help remove them.

The purpose of this study is to evaluate the effectiveness and safety of ALXN2220 in adults with ATTR-CM. Participants in the study will receive either the ALXN2220 treatment or a placebo, which is a substance with no active medication. The study will be conducted over a period of time, during which participants will receive regular injections of the treatment or placebo. Throughout the study, participants will be monitored for any changes in their heart condition and overall health.

The study aims to determine if ALXN2220 can help reduce the symptoms and progression of ATTR-CM by comparing the health outcomes of those receiving the treatment to those receiving the placebo. Participants will undergo various health assessments, including heart function tests and physical examinations, to track their progress. The study will provide valuable information on whether ALXN2220 is a safe and effective treatment option for people with ATTR-CM.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization

    Upon joining the study, participants are randomly assigned to receive either the ALXN2220 medication or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

  2. Step 2

    Treatment administration

    Participants receive the assigned treatment as a solution for infusion. The specific dosage and frequency are determined by the study protocol and are administered under medical supervision.

  3. Step 3

    Blinded treatment period

    During this period, the effectiveness and safety of ALXN2220 are evaluated. Participants continue to receive their assigned treatment and undergo regular assessments to monitor their health and any potential side effects.

  4. Step 4

    Primary endpoint evaluation

    The main goal is to assess the difference in the occurrence of cardiovascular events and overall health status between the ALXN2220 and placebo groups. This includes monitoring for any adverse events and changes in health indicators.

  5. Step 5

    Secondary endpoint evaluation

    Additional outcomes are measured, such as changes in physical function and quality of life over a 24-month period, as well as the incidence of any treatment-related adverse effects.

  6. Step 6

    Study completion

    The study is expected to conclude by October 31, 2028. Participants will have contributed valuable data to assess the potential benefits and risks of ALXN2220 for treating transthyretin amyloid cardiomyopathy.

Who can join the trial?

10 criteria

  • Must be a male or female aged between 18 and 90 years old.
  • If using a treatment for ATTR amyloidosis, the dose must be stable for at least 90 days before the study starts.
  • Participants must use contraception according to local rules. Women must not be pregnant or breastfeeding and should use effective birth control if they can have children. Men must agree to use contraception and not donate sperm.
  • Must have a confirmed diagnosis of **ATTR-CM** (a type of heart condition) with specific tests showing heart involvement.
  • The thickness of the heart's septal wall must be 12 mm or more, as measured by an ultrasound of the heart.
  • Must have a **NT-proBNP** level greater than 2000 pg/mL, which is a blood test that helps assess heart function.
  • Must have been treated with a **loop diuretic** (a type of medication that helps remove excess fluid) for at least 30 days before the study.
  • Must have a history of heart failure, shown by events like hospitalization for heart failure, urgent visits for heart failure, or episodes of fluid overload.
  • Must be classified as **NYHA Class II-IV**, which describes the severity of heart failure symptoms.
  • Must have a life expectancy of at least 6 months, as judged by the study doctor.

Who cannot join the trial?

3 criteria

  • Patients who are not adults cannot participate.
  • Patients who do not have Transthyretin Amyloid Cardiomyopathy (ATTR-CM) cannot participate. ATTR-CM is a condition where a protein called transthyretin builds up in the heart, affecting its function.
  • Patients who are part of a vulnerable population, such as those who cannot make decisions for themselves, cannot participate.
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Investigated drugs

ALXN2220 is a medication being studied for its potential to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This condition involves the buildup of abnormal protein deposits in the heart, which can lead to heart problems. ALXN2220 is designed to help reduce these protein deposits, potentially improving heart function and reducing related health issues. The study aims to evaluate how effective and safe ALXN2220 is for patients with this condition.

What is already known about the treatment

ALXN2220 – ALXN2220 is administered intravenously and is currently being studied in clinical trials for its effectiveness in treating adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM). This medication is in the investigational stage, with ongoing research to determine its safety and efficacy. The main therapeutic indication for ALXN2220 is to reduce amyloid deposits in the heart, which are characteristic of ATTR-CM. At the molecular level, ALXN2220 works by targeting and depleting amyloid fibrils, potentially improving heart function and reducing related cardiovascular events. It is classified pharmacologically as an amyloid depleter.

Investigated diseases

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) – This is a rare heart condition caused by the buildup of abnormal protein deposits called amyloid in the heart tissue. These deposits can lead to the stiffening of the heart muscle, making it difficult for the heart to pump blood effectively. Over time, this can result in symptoms such as fatigue, shortness of breath, and swelling in the legs and feet. The disease typically progresses slowly, but the accumulation of amyloid can eventually lead to more severe heart problems. ATTR-CM is often seen in older adults and can be hereditary or occur without a family history. The condition requires careful monitoring to manage its impact on heart function.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-506669-70-00Protocol codeALXN2220-ATTRCM-301Estimated enrolment1 000 patientsSponsorAlexion Pharmaceuticals Inc.

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