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Ongoing Clinical Trials for Primary Amyloidosis

There are currently 6 clinical trials ongoing for primary amyloidosis, also known as AL amyloidosis or immunoglobulin light-chain amyloidosis. These trials are testing various treatment combinations across multiple European countries and Israel, including medications such as teclistamab, daratumumab, birtamimab, and several chemotherapy agents. The trials focus on both newly diagnosed patients and those with relapsed or advanced disease.

Clinical trial locations

Study of Teclistamab for Patients with Previously Treated AL Amyloidosis

This trial is testing a medication called teclistamab in patients who have already received treatment for their condition. Teclistamab is a type of bispecific T-cell engager that helps the immune system target and destroy abnormal cells responsible for producing the harmful proteins that build up in organs.

Main inclusion criteria: Participants must be at least 18 years old with a confirmed diagnosis based on specific tissue tests. They need to have Mayo stage I-IIIA cardiac disease, meaning the heart is affected but not at the most advanced stage. Those joining must have relapsed disease with at least one previous treatment including specific drugs like daratumumab and bortezomib, completing at least two cycles. The disease must be measurable through blood tests, with symptoms in at least one organ such as the heart, kidneys, liver, digestive system, or nerves. Patients must have stopped other treatments for at least 14 days before starting this trial.

Main exclusion criteria: Patients who have never received treatment for this condition cannot participate. Those outside the specified age range or not meeting the clinical trial group criteria are also excluded.

Trial focus: The main goal is to evaluate how well teclistamab works after three cycles of treatment. Researchers will monitor whether patients achieve a complete response, meaning the disease is no longer detectable in the blood. They will also assess how affected organs respond and track any side effects throughout the study.

Investigational drug: Teclistamab is given as an injection under the skin. It works by targeting specific proteins on immune cells to help the body attack and destroy abnormal cells producing harmful light chain proteins.

Study on the Safety and Effectiveness of Linvoseltamab, Sarilumab, and Dexamethasone in Adults with Relapsed or Refractory AL Amyloidosis

This trial evaluates linvoseltamab in patients whose disease has returned or has not responded to previous treatments. Linvoseltamab is a monoclonal antibody designed to target specific molecules involved in the disease process.

Main inclusion criteria: Participants must have a confirmed diagnosis with measurable disease tracked through blood protein levels. They must have received at least one but no more than four previous therapies, which may include autologous stem cell transplant. The treating physician must determine that further treatment is needed. Blood tests showing NT-proBNP levels of 8500 ng/L or less are required, along with adequate liver, blood, kidney, and heart function. An ECOG performance score of 2 or less is necessary, indicating patients can perform most daily activities with some limitations.

Main exclusion criteria: Patients with conditions other than relapsed or refractory systemic light chain amyloidosis, those outside the specified age range, vulnerable populations requiring special protection, and those not meeting specific health requirements cannot participate.

Trial focus: The study aims to find the best dose of linvoseltamab for future research and to determine how well it achieves a complete response in the blood. The trial has two phases: the first assesses safety and tolerability, while the second evaluates effectiveness. Some participants may receive a placebo for comparison.

Investigational drug: Linvoseltamab is given intravenously directly into a vein. It targets specific proteins to reduce their harmful effects and is being studied alongside additional medications like dexamethasone to manage side effects.

Study of Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone for Patients with Newly Diagnosed AL Amyloidosis with Heart Involvement

This trial tests a combination of four medications in patients newly diagnosed with the condition when the heart is affected. The combination includes daratumumab, bortezomib, cyclophosphamide, and dexamethasone.

Main inclusion criteria: Participants must be at least 18 years old with a new diagnosis confirmed through specific medical tests. Heart involvement is required, with or without other organs affected. An ECOG Performance Status score of 0, 1, or 2 is needed. Blood test results must fall within specific ranges, including hemoglobin levels of at least 8.0 g/dL, platelet count of at least 50,000 per microliter, and kidney function with an estimated glomerular filtration rate of at least 20 mL/min/1.73 m². Women who can have children must have a negative pregnancy test and agree to use effective birth control. Men must use condoms during the study and for specified periods afterward.

Main exclusion criteria: Patients with other types of amyloidosis, those without newly diagnosed systemic disease or cardiac involvement, those outside the specified age range, those unable to provide informed consent, pregnant or breastfeeding patients, and those with other serious health conditions are excluded.

Trial focus: The study examines the safety of these treatments, particularly concerning heart health. It aims to identify strategies to reduce heart-related side effects and understand how daratumumab behaves in the body, especially among racial and ethnic minorities. Researchers will monitor heart-related events, response rates, and overall survival.

Investigational drugs: Daratumumab helps the immune system target abnormal cells. Bortezomib prevents protein breakdown in cells, leading to abnormal cell death. Cyclophosphamide slows or stops abnormal cell growth. Dexamethasone reduces inflammation and enhances the effects of other medications.

Study on Birtamimab and Bortezomib for Patients with Advanced Light Chain Amyloidosis

This trial evaluates birtamimab combined with standard treatment including bortezomib in patients with Mayo Stage IV disease, the most advanced form affecting the heart severely.

Main inclusion criteria: Participants must be at least 18 years old with a new diagnosis and no previous treatment. Heart involvement is required, confirmed through specific blood tests showing NT-proBNP levels of 1800 pg/mL or higher, Troponin-T levels of 0.025 ng/mL or higher, and dFLC levels of 18 mg/dL or higher. Patients must be planning to start first-line chemotherapy with weekly bortezomib injections under the skin.

Main exclusion criteria: Patients with other serious medical conditions, recent heart attacks or severe heart problems, severe kidney disease, pregnant or breastfeeding women, those who participated in another trial within the last 30 days, those unable to follow study procedures, those with allergies to study medications, active infections requiring treatment, and those with a history of drug or alcohol abuse are excluded.

Trial focus: The study has two phases: a double-blind phase where neither participants nor researchers know who receives the actual treatment or placebo, and an open-label extension phase where all participants receive birtamimab to evaluate long-term safety. The main outcome is time to death during the double-blind phase, with additional assessments of physical abilities and quality of life over nine months.

Investigational drug: Birtamimab is given through an intravenous infusion. It is a monoclonal antibody that targets and binds to amyloid proteins, potentially helping clear these deposits from tissues and improving survival.

Study on Daratumumab, Cyclophosphamide, Bortezomib, and Dexamethasone for Patients with Newly Diagnosed Systemic AL Amyloidosis

This trial compares two treatment approaches in newly diagnosed patients: one group receives daratumumab combined with cyclophosphamide, bortezomib, and dexamethasone, while the other receives the three-drug combination without daratumumab.

Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis through specific tissue tests. The disease must be measurable through blood tests. At least one organ must be affected. Physical health status should allow performance of daily activities with some limitations. Blood test results must meet specific criteria including adequate white blood cells, red blood cells, platelets, and normal liver and kidney function. Women who can have children must have a negative pregnancy test and agree to use reliable birth control. Men must agree to use birth control methods if sexually active with women who can have children.

Main exclusion criteria: Patients with different types of amyloidosis, those who already received treatment, those with serious health conditions, pregnant or breastfeeding women, those unable to follow study procedures, and those with allergies to study medications are excluded.

Trial focus: The study will last up to 36 months with regular treatments and monitoring. The primary goal is to determine the overall complete hematologic response rate, evaluating how well treatment reduces abnormal protein levels in the blood. Researchers will compare the two groups to see which is more effective.

Investigational drugs: Daratumumab targets abnormal cells through the immune system. Cyclophosphamide is a chemotherapy drug that slows abnormal cell growth. Bortezomib prevents protein breakdown in cells, leading to abnormal cell death. Dexamethasone reduces inflammation and enhances other medications’ effects.

Study on the Safety and Effectiveness of Sargramostim with Daratumumab, Cyclophosphamide, Bortezomib, and Dexamethasone in Untreated Light Chain Amyloidosis Patients

This trial tests a combination therapy called D-VCd (daratumumab, cyclophosphamide, bortezomib, and dexamethasone) together with sargramostim in patients who have not yet received treatment.

Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis through tissue tests. The disease must be measurable through blood protein levels. One or more organs must be affected. Performance status should be 0, 1, or 2 on the ECOG scale, meaning patients can perform daily activities with some limitations. Blood test results must meet specific criteria for white blood cells, red blood cells, and platelets. Women who can have children must have a negative pregnancy test and agree to use reliable birth control. Men must agree to use birth control if sexually active with women who can have children.

Main exclusion criteria: Patients with other types of amyloidosis, those who received previous treatment within a certain period, those with severe unstable heart or kidney problems, active infections, another type of cancer within the last five years, pregnant or breastfeeding women, those with known allergies to study medications, and those who participated in another trial recently are excluded.

Trial focus: The study evaluates how effective and safe this combination is in achieving a complete blood response and improving organ function. Researchers will monitor participants’ response to treatment and any side effects. The goal is to determine if this combination can reduce amyloid deposits and improve organ function.

Investigational drugs: Sargramostim stimulates white blood cell production and may help break down amyloid deposits. Daratumumab targets and destroys specific immune system cells involved in the disease. Cyclophosphamide slows cancer cell growth. Bortezomib interferes with cancer cell growth. Dexamethasone reduces inflammation and suppresses the immune system.

Summary

The six ongoing clinical trials for primary amyloidosis reflect a comprehensive research effort across Europe and Israel. A notable pattern is the concentration of trials in countries such as Germany, France, Italy, Greece, and the Netherlands, with multiple studies available in each location. This geographic distribution provides patients in these regions with greater access to experimental treatments.

The trials predominantly focus on daratumumab, which appears in four of the six studies, highlighting its importance in current research approaches. This medication is being tested both in newly diagnosed patients and those with relapsed or advanced disease. Combination therapies are the standard approach, with most trials pairing multiple medications to target the disease more effectively.

The trials address different patient populations: some focus on newly diagnosed patients, while others target those with relapsed, refractory, or advanced disease including Mayo Stage IV. This range ensures that research covers the full spectrum of disease stages. Heart involvement is a particular focus, with several trials specifically examining cardiac safety and outcomes.

The investigational medications include both established treatments like bortezomib, cyclophosphamide, and dexamethasone, as well as newer approaches such as teclistamab, linvoseltamab, birtamimab, and sargramostim. This combination of proven and experimental therapies reflects the ongoing evolution of treatment strategies for this challenging condition.

Ongoing Clinical Trials on Primary amyloidosis

  • Study on Daratumumab, Cyclophosphamide, Bortezomib, and Dexamethasone for Patients with Newly Diagnosed Systemic AL Amyloidosis

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Denmark France Germany Greece Hungary +5

  • Study on Birtamimab and Bortezomib for Patients with Advanced Light Chain Amyloidosis

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark France Germany +8

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