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Study on the Effectiveness and Safety of CAEL-101 with Drug Combination for Patients with Advanced AL Amyloidosis

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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 called AL amyloidosis, specifically in patients with a severe form known as Stage IIIb cardiac AL amyloidosis. This disease involves the buildup of abnormal proteins in the heart, which can lead to heart problems. The study is testing a new treatment called CAEL-101, also known by its code name anselamimab. This treatment is being compared to a placebo, which is a substance with no active medication, to see if it can improve the survival of patients who have not received any previous treatment for this condition.

Participants in the study will receive either the new treatment or a placebo, along with standard treatments for a related condition called plasma cell dyscrasia. The standard treatments include medications such as cyclophosphamide, bortezomib, and dexamethasone. These medications are commonly used to treat disorders involving abnormal plasma cells, which are a type of white blood cell. The study aims to determine if the combination of CAEL-101 and these standard treatments can help patients live longer and improve their quality of life.

The study will be conducted over a period of time, during which participants will receive their assigned treatments and be monitored for any changes in their health. The main goal is to assess the time it takes for any cause of death to occur from the start of the study. Additionally, the study will look at other health measures, such as heart function and physical activity levels, to evaluate the overall effectiveness and safety of the new treatment. Participants will be closely observed to ensure their safety throughout the study.

The research process

The trial runs in 5 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 investigational drug CAEL-101 or a placebo, in addition to the standard treatment for plasma cell dyscrasia, which includes cyclophosphamide, bortezomib, and dexamethasone.

  2. Step 2

    Treatment administration

    The standard treatment regimen, known as CyBorD, consists of cyclophosphamide, bortezomib, and dexamethasone. Cyclophosphamide and bortezomib are administered intravenously, while dexamethasone is taken orally.

    The investigational drug CAEL-101 or placebo is administered intravenously. The frequency and dosage of these medications will be determined by the study protocol and the healthcare team.

  3. Step 3

    Monitoring and evaluation

    Throughout the study, the patient's health and response to treatment will be closely monitored. This includes regular assessments of heart function, as the study focuses on patients with stage IIIb cardiac AL amyloidosis.

    The primary goal is to evaluate the effectiveness of CAEL-101 in improving overall survival and its safety when combined with the standard treatment.

  4. Step 4

    Study duration

    The study is expected to continue until December 2026. During this period, patients will undergo various assessments to measure changes in their condition, including heart function and physical capabilities.

    Key assessments will occur at baseline and continue through week 50, focusing on metrics such as the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS), Global Longitudinal Strain (GLS%), and the 6-Minute Walk Test (6MWT) distance.

  5. Step 5

    End of study

    At the end of the study, the data collected will be analyzed to determine the impact of CAEL-101 on patient outcomes compared to the placebo.

    Patients who complete the study will have their last known health status recorded, and those who discontinue early will still be included in the analysis based on their last recorded data.

Who can join the trial?

7 criteria

  • The patient must have **Stage IIIb AL amyloidosis**, which is a specific stage of a condition where abnormal proteins build up in organs, affecting the heart.
  • The patient must have a measurable blood condition at the time of screening, which can be shown by specific blood tests.
  • The patient must have a confirmed diagnosis of amyloidosis through a special test that looks at tissue samples under a microscope.
  • The patient must have heart involvement, meaning the heart is affected by amyloidosis, shown by specific tests like a heart biopsy, an ultrasound of the heart, or a special heart scan called an MRI.
  • The patient must be planning to start a specific treatment for a related condition called plasma cell dyscrasia, using a combination of drugs known as CyBorD.
  • Women who can have children must have a negative pregnancy test and agree to use effective birth control during the study and for a certain time after.
  • Men must either be unable to father children or agree to use effective birth control and not donate sperm during the study and for a certain time after.

Who cannot join the trial?

10 criteria

  • Patients with other serious health conditions that could interfere with the study.
  • Patients who have received certain treatments or medications that might affect the study results.
  • Patients who are unable to follow the study procedures or attend required visits.
  • Patients who are pregnant or breastfeeding.
  • Patients with a history of allergic reactions to the study medication.
  • Patients who have participated in another clinical trial recently.
  • Patients with certain infections or diseases that could impact the study.
  • Patients with a history of substance abuse that could interfere with the study.
  • Patients who have had a recent major surgery.
  • Patients with mental health conditions that might affect their ability to participate.
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Investigated drugs

  • CAEL-101

    is an investigational medication being studied for its potential to improve overall survival in patients with a specific type of amyloidosis known as AL amyloidosis. This medication is being tested in combination with standard treatments for plasma cell dyscrasia, a condition related to abnormal plasma cells, to see if it can provide additional benefits to patients who have not yet received any treatment for their condition.

  • Plasma Cell Dyscrasia Treatment

    refers to the standard therapies used to manage conditions caused by abnormal plasma cells, such as AL amyloidosis. These treatments aim to control the disease and improve patient outcomes. In this study, the effectiveness and safety of these treatments are being evaluated both alone and in combination with CAEL-101.

What is already known about the treatment

CAEL-101 – This medication is administered intravenously and is currently being studied in clinical trials for its potential use in treating stage IIIb cardiac AL amyloidosis. It is not yet widely recognized in medical literature as a standard treatment. The main therapeutic indication is to improve overall survival in patients with this specific type of amyloidosis. CAEL-101 works by targeting and binding to amyloid fibrils, which may help in clearing these deposits from the heart tissue. It is classified as a monoclonal antibody, a type of protein designed to interact with specific targets in the body.

Investigated diseases

Cardiac AL Amyloidosis – This disease is characterized by the buildup of abnormal protein deposits, known as amyloid, in the heart tissue. These deposits can interfere with the heart's normal function, leading to symptoms such as fatigue, shortness of breath, and swelling in the legs. As the condition progresses, the heart may become stiffer, making it harder for it to pump blood effectively. This can result in heart failure and other complications related to reduced cardiac output. The disease is often associated with a type of blood cell disorder called plasma cell dyscrasia. Stage IIIb indicates an advanced stage where the heart is significantly affected, impacting the patient's daily activities and overall health.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-503073-11-00Protocol codeCAEL101-301Estimated enrolment125 patientsSponsorAlexion Pharmaceuticals Inc.

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