Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study on the Effectiveness and Safety of CAEL-101 with Plasma Cell Dyscrasia Treatment in Patients with Stage IIIa AL Amyloidosis

Fast replyRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

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 IIIa cardiac AL amyloidosis. This disease involves the buildup of abnormal proteins in organs and tissues, which can affect the heart. The study is testing a new treatment called CAEL-101, which is given as a solution through an intravenous infusion. The trial will compare the effects of CAEL-101 combined with standard treatment for a related condition called plasma cell dyscrasia against a placebo combined with the same standard treatment.

The purpose of the study is to see if CAEL-101 can help improve the survival of patients who have not yet received treatment for their condition. The study will also look at how safe and tolerable CAEL-101 is when used with the standard treatment. Participants will receive either the new treatment or a placebo, along with their regular therapy, and will be monitored over time to assess their health and any changes in their condition.

Throughout the study, participants will undergo various health checks, including monitoring of vital signs and heart function, to ensure their safety and to gather information on how the treatment affects their condition. The study aims to provide valuable insights into the potential benefits of CAEL-101 for patients with this challenging disease.

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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and specific tests to confirm the diagnosis of stage IIIa cardiac AL amyloidosis.

    Tests may include blood tests, imaging studies, and a biopsy to confirm the presence of amyloid deposits.

  2. Step 2

    Treatment initiation

    The treatment phase begins with the administration of the study drug CAEL-101 or a placebo. This is given through an intravenous infusion.

    The standard treatment for plasma cell dyscrasia, known as the CyBorD regimen (cyclophosphamide, bortezomib, and dexamethasone), is also administered.

  3. Step 3

    Treatment schedule

    The study drug or placebo is administered regularly, with the frequency and dosage determined by the study protocol.

    The CyBorD regimen is administered according to the standard of care guidelines.

  4. Step 4

    Monitoring and follow-up

    Regular monitoring is conducted to assess the effectiveness and safety of the treatment. This includes physical examinations, blood tests, and heart function tests.

    Participants are monitored for any side effects or adverse reactions to the treatment.

  5. Step 5

    Evaluation of outcomes

    The primary outcomes being evaluated include overall survival and the frequency of hospitalizations due to heart-related issues.

    Secondary outcomes include changes in heart function and quality of life assessments over a period of 50 weeks.

  6. Step 6

    Completion of study

    The study is expected to conclude by August 2027. Participants will have a final assessment to evaluate the long-term effects of the treatment.

    Data collected will contribute to understanding the safety and effectiveness of CAEL-101 in treating AL amyloidosis.

Who can join the trial?

16 criteria

  • The patient must have **stage IIIa 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 specific level of a heart-related protein called **NT-proBNP** that is 650 ng/L or higher during the screening process.
  • The patient must have a measurable blood condition, which can be shown by one of the following:
    • A difference in certain blood proteins called **free light chains** greater than 4 mg/dL.
    • An abnormal level of a specific free light chain protein with an unusual ratio of two proteins called Kappa and Lambda.
    • A specific protein level in the blood called **m-spike** greater than 0.5 g/dL.
    • The patient must have a confirmed diagnosis of amyloidosis, which is a condition where abnormal proteins build up in tissues, shown by:
      • A special test called **polarizing light microscopy** on tissue samples stained with Congo red.
      • Confirmation of AL amyloid deposits using one of the following methods:
        • **Immunohistochemistry** or **Immunofluorescence** tests.
        • **Mass spectrometry**, a detailed analysis method.
        • **Electron microscopy**, a type of imaging that shows detailed structures.
        • The patient must have heart involvement, shown by:
          • Signs and symptoms of heart failure with a confirmed diagnosis of AL amyloidosis and no other reason for heart failure.
          • One of the following:
            • A heart tissue test called **endomyocardial biopsy** showing AL cardiac amyloidosis.
            • An **echocardiogram** showing a specific heart wall thickness greater than 12 mm without other causes like high blood pressure or valve problems.
            • A heart scan called **cardiac MRI** with a special contrast agent showing cardiac amyloidosis.
            • The patient must be planning to start a specific first-line treatment for a condition called **plasma cell dyscrasia** using a combination of drugs: cyclophosphamide, bortezomib, and dexamethasone, known as the **CyBorD regimen**.
            • Women who can have children must have a negative pregnancy test during screening and agree to use highly effective birth control from screening until at least 5 months after the last study drug or 12 months after the last dose of their plasma cell dyscrasia therapy, whichever is longer.
            • Men must be surgically sterile or agree to use highly effective birth control and not donate sperm from screening until at least 5 months after the last study drug or 12 months after the last dose of their plasma cell dyscrasia therapy, whichever is longer.

Who cannot join the trial?

10 criteria

  • Patients who have already received treatment for their condition cannot participate.
  • Patients with other serious health problems that could interfere with the study are not eligible.
  • Pregnant or breastfeeding women are excluded from the study.
  • Patients who are unable to follow the study procedures or attend required visits cannot join.
  • Individuals with a history of allergic reactions to similar treatments are not allowed to participate.
  • Patients with certain infections or diseases that could affect the study results are excluded.
  • Anyone who is currently participating in another clinical trial is not eligible.
  • Patients with a history of substance abuse that could interfere with the study are not allowed to join.
  • Individuals who have had a recent major surgery may be excluded from the study.
  • Patients with certain heart conditions that could pose a risk during the study are not eligible.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

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

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • CAEL-101

    is an investigational medication being studied for its potential to improve overall survival and reduce the frequency of cardiovascular hospitalizations in patients with a specific type of amyloidosis known as AL amyloidosis. This medication is being tested in patients who have not previously received treatment for their condition. The study aims to assess the safety and effectiveness of CAEL-101 when used alongside standard treatments for plasma cell dyscrasia.

  • Plasma Cell Dyscrasia Treatment

    refers to the standard therapies used to manage conditions related to abnormal plasma cells, such as AL amyloidosis. These treatments aim to control the underlying disease by targeting the abnormal plasma cells, thereby reducing the production of amyloid proteins that can cause organ damage. The study is evaluating how well these treatments work in combination with CAEL-101.

What is already known about the treatment

CAEL-101 – CAEL-101 is administered intravenously and is currently being studied in clinical trials for its potential use in treating stage IIIa cardiac AL amyloidosis. This medication is in the investigational stage, with ongoing research to determine its efficacy and safety. It is primarily indicated for patients with this specific type of amyloidosis, aiming to improve overall survival and reduce cardiovascular hospitalizations. CAEL-101 works by targeting amyloid deposits in the heart, potentially reducing their accumulation and associated damage. It is classified as a monoclonal antibody, which is a type of protein designed to bind to specific targets in the body.

Investigated diseases

Cardiac AL Amyloidosis – This is a condition where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. These deposits can interfere with the heart's normal function, leading to issues such as heart failure. In stage IIIa, the disease is more advanced, and symptoms may include fatigue, swelling in the legs, and shortness of breath. The progression involves worsening heart function as the amyloid deposits increase. Over time, this can lead to more severe symptoms and complications related to the heart's ability to pump blood effectively. The condition is considered rare and requires specialized medical attention.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-503072-84-00Protocol codeCAEL101-302Estimated enrolment281 patientsSponsorAlexion Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

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).