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Study on Birtamimab and Bortezomib for Patients with Advanced Light Chain 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 known as Light Chain (AL) Amyloidosis, which is a rare disease where abnormal proteins build up in organs and tissues, affecting their normal function. The study will evaluate the effectiveness and safety of a treatment involving a medication called Birtamimab, which is a type of protein designed to target and neutralize harmful substances in the body. Birtamimab will be used in combination with the standard treatment for AL amyloidosis, which includes medications like Bortezomib, a drug that helps to slow down or stop the growth of abnormal cells. The study will also involve a comparison with a placebo, which is an inactive substance used to assess the treatment's true effects.

The purpose of the study is to determine how well Birtamimab, when added to the standard treatment, works in patients with a severe form of AL amyloidosis, known as Mayo Stage IV. Participants will receive the treatment through an intravenous infusion, which means the medication is delivered directly into the bloodstream. The study will be conducted in two phases: a double-blind phase, where neither the participants nor the researchers know who is receiving the actual treatment or the placebo, and an open-label extension phase, where all participants will receive Birtamimab to evaluate its long-term safety.

Throughout the study, researchers will monitor the participants' health and track important outcomes, such as the time it takes for any cause of death to occur during the double-blind phase. Additionally, they will assess changes in physical abilities and quality of life over a period of nine months. This trial aims to provide valuable insights into the potential benefits of Birtamimab for patients with AL amyloidosis, offering hope for improved treatment options in the future.

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

    Joining the study

    Upon joining the study, you will be randomly assigned to one of two groups. One group will receive the medication birtamimab along with standard care, while the other group will receive a placebo with standard care. This process is double-blind, meaning neither you nor the study team will know which group you are in.

  2. Step 2

    Initial assessment

    You will undergo an initial assessment to confirm your eligibility. This includes confirming your diagnosis of light chain (AL) amyloidosis and ensuring you meet specific health criteria.

  3. Step 3

    Treatment phase

    During the treatment phase, you will receive birtamimab or a placebo through an intravenous infusion. This means the medication will be administered directly into your vein. The frequency and dosage will be determined by the study protocol.

    In addition to the study medication, you will receive standard care, which includes bortezomib administered as a subcutaneous injection once a week. This is a common treatment for your condition.

  4. Step 4

    Monitoring and follow-up

    Throughout the study, your health will be closely monitored. This includes regular check-ups and tests to assess your response to the treatment and to ensure your safety.

    You will be asked to participate in assessments such as the 6-minute walk test (6MWT) and complete questionnaires about your physical health.

  5. Step 5

    Open-label extension phase

    After the initial treatment phase, you may have the opportunity to enter an open-label extension phase. In this phase, all participants will receive birtamimab along with standard care, regardless of the group they were initially assigned to.

    The purpose of this phase is to evaluate the long-term safety of the medication.

  6. Step 6

    Completion of the study

    The study is expected to conclude by June 2027. At the end of the study, you will have a final assessment to evaluate your overall health and the effects of the treatment.

Who can join the trial?

6 criteria

  • Must be at least 18 years old and of legal age to give consent according to local laws.
  • Must have a new diagnosis of AL amyloidosis and not have received any treatment for it before.
  • Must have heart involvement due to AL amyloidosis.
  • Must have a confirmed diagnosis of AL amyloidosis.
  • Must have Mayo Stage IV AL Amyloidosis, which is determined by specific blood test results: NT-proBNP (a heart-related protein) levels of 1800 pg/mL or higher, and Troponin-T (a heart-related protein) levels of 0.025 ng/mL (or 40 ng/L for high sensitivity tests) or higher, and dFLC (a type of protein in the blood) levels of 18 mg/dL or higher.
  • Must have a plan to start first-line chemotherapy that includes bortezomib, which is given as a weekly injection under the skin.

Who cannot join the trial?

10 criteria

  • Patients with any other serious medical condition that might interfere with the study.
  • Patients who have had a recent heart attack or severe heart problems.
  • Patients with severe kidney disease.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial within the last 30 days.
  • Patients who are unable to follow the study procedures.
  • Patients with a history of allergic reactions to the study medication.
  • Patients with active infections that require treatment.
  • Patients with a history of drug or alcohol abuse.
  • Patients who have received certain medications that might interfere with the study.
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Investigated drugs

Birtamimab is a medication being studied for its potential to help people with a condition called AL amyloidosis. This condition causes abnormal proteins to build up in organs, which can lead to serious health problems. Birtamimab is given through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein. The goal of this medication is to improve the survival of patients by reducing the harmful effects of these protein deposits in the body. In this clinical trial, birtamimab is being tested to see if it can help patients live longer and improve their quality of life when used alongside standard treatments for AL amyloidosis.

What is already known about the treatment

Birtamimab – Birtamimab is administered intravenously, meaning it is given directly into a vein through an injection. It is currently being studied in clinical trials for its effectiveness and safety in treating a condition called Light Chain (AL) Amyloidosis, particularly in patients at an advanced stage of the disease. The main therapeutic goal of birtamimab is to improve survival rates by reducing the harmful effects of amyloid deposits in the body. At the molecular level, birtamimab works by targeting and binding to the amyloid proteins, which may help in clearing these deposits from tissues. It is classified as a monoclonal antibody, a type of medication designed to specifically target certain proteins in the body.

Investigated diseases

Light Chain (AL) Amyloidosis – This disease is characterized by the abnormal deposition of amyloid proteins, specifically light chains, in various tissues and organs. These deposits can disrupt normal function, leading to organ damage over time. The progression of AL amyloidosis often involves the heart, kidneys, liver, and nervous system. As the disease advances, it can cause symptoms such as fatigue, weight loss, and swelling due to organ dysfunction. The accumulation of amyloid proteins can lead to complications, affecting the quality of life. The rate of progression can vary significantly among individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511066-36-00Protocol codeNEOD001-301Estimated enrolment220 patientsSponsorProthena Biosciences Limited

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