Not recruitingRare disease

Study on the Effects and Safety of ALXN2220 for Adults with Transthyretin Amyloid Cardiomyopathy

Verified siteInvestigationalNo 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 heart condition known as Transthyretin Amyloid Cardiomyopathy (ATTR-CM). ATTR-CM is a disease where abnormal proteins, called amyloids, build up in the heart, affecting its function. The study will use a 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, targeting the amyloids involved in ATTR-CM.

The purpose of this study is to evaluate how the body responds to re-treatment with ALXN2220 and to assess its safety in patients with ATTR-CM. Participants in the study will receive ALXN2220 as a solution for injection. The study will monitor changes in the heart using imaging techniques like cMRI (cardiac magnetic resonance imaging) and scintigraphy, which are methods to visualize the heart and assess its condition. The study will also track any side effects and changes in vital signs, such as blood pressure and heart rate, as well as laboratory test results and heart electrical activity through ECG (electrocardiogram).

Throughout the study, the levels of ALXN2220 in the blood will be measured, and any immune response to the medication will be monitored. The study aims to provide valuable information on the effectiveness and safety of ALXN2220 for patients with ATTR-CM, potentially leading to improved treatment options for this condition.

The research process

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

Step 1
Initial visit
Upon joining the study, the first step involves an initial visit to the clinical trial site. During this visit, a healthcare professional will review your medical history and confirm your eligibility to participate in the study.
A blood test will be conducted to ensure that women of childbearing potential have a negative pregnancy test. This is a necessary step to ensure safety during the trial.
Step 2
Informed consent
You will be asked to read and sign an informed consent form. This document explains the details of the study, including potential risks and benefits. Signing this form indicates that you understand the study and agree to participate.
Step 3
Treatment administration
The study involves re-treatment with a medication called ALXN2220, which is a recombinant human anti-ATTR immunoglobulin G1 (IgG1) monoclonal antibody. This medication is administered as a solution for infusion.
The frequency and dosage of the medication will be determined by the study protocol and administered by healthcare professionals at the clinical trial site.
Step 4
Regular monitoring
Throughout the study, regular visits to the clinical trial site will be required. During these visits, healthcare professionals will monitor your health and the effects of the medication.
Tests such as cMRI (cardiac magnetic resonance imaging) and scintigraphy will be conducted to assess changes from the baseline to Visit 16. These tests help evaluate the impact of the treatment on your condition.
Step 5
Safety assessments
Safety assessments will be conducted regularly to monitor for any side effects or adverse events. This includes checking vital signs, conducting physical examinations, and performing laboratory tests.
The incidence of TEAEs (treatment-emergent adverse events), including serious TEAEs, will be recorded and evaluated.
Step 6
Study completion
Upon completion of the study, a final visit will be scheduled. During this visit, a comprehensive evaluation will be conducted to assess your overall health and the outcomes of the treatment.
You will be informed about the results of the study and any further steps, if necessary.

Who can join the trial?

6 criteria

Must be an adult aged 18 years or older.
Must have a condition called Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
Must have received at least one dose of the study drug called ALXN2220 in a previous study and tolerated it well, according to the study doctor.
Must be able to understand and sign a consent form before starting any study procedures, and be willing and able to follow all study procedures.
Women who can have children must have a negative pregnancy test before starting the study and agree to use highly effective birth control methods approved by a doctor from the start of the study until 5 months after the study ends.
Men must agree to not donate semen during the study and for at least 7 months after the last dose of the study drug. They must either abstain from heterosexual intercourse or use a condom during intercourse, and it is advised that their female partner also uses a highly effective birth control method.

Who cannot join the trial?

3 criteria

Patients who do not have Transthyretin Amyloid Cardiomyopathy (ATTR-CM) cannot participate.
Patients who are not adults cannot participate.
Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups like children, pregnant women, or people who cannot make decisions for themselves.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Centre Hospitalier Universitaire De Rennes
Rennes, France
Verified
Hopital Beaujon
Clichy, France
Verified
Hospital Universitario Puerta De Hierro De Majadahonda
Majadahonda, Spain

Trial locations

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Investigated drugs

ALXN2220 is a medication being studied for its effects on a condition called transthyretin amyloid cardiomyopathy. This condition affects the heart and can lead to heart failure. The medication is being tested to see how it works in the body and to check its safety when given to patients who have already been treated with it before. The goal is to understand how the medication can help manage the disease and improve heart function in patients. The study is focused on understanding the medication's effects and ensuring it is safe for patients to use again.

Alxn2220

What is already known about the treatment

ALXN2220 – ALXN2220 is administered as an injection, typically given under the skin or into a vein. It is currently being studied in clinical trials, specifically in a Phase 2 study, to understand its effects and safety when used again in patients with a heart condition called Transthyretin Amyloid Cardiomyopathy (ATTR-CM). This medication is being explored for its potential to help manage this condition by targeting the underlying causes at a molecular level, although the exact mechanism is still under investigation. ALXN2220 falls under the category of investigational drugs, meaning it is not yet approved for general medical use and is still being tested for its effectiveness and safety.

Investigated diseases

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) – Transthyretin Amyloid Cardiomyopathy is a condition where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. This buildup occurs due to misfolded transthyretin proteins, which are normally responsible for transporting thyroid hormones and vitamin A. Over time, the amyloid deposits cause the heart walls to thicken and stiffen, leading to impaired heart function. As the disease progresses, the heart's ability to pump blood effectively is reduced, which can lead to symptoms such as fatigue, shortness of breath, and swelling in the legs. The condition is progressive, meaning it worsens over time, and it primarily affects older adults. ATTR-CM is considered a rare disease, with genetic and age-related factors contributing to its development.