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

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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 Light Chain Amyloidosis, which is a rare disease where abnormal proteins called amyloids build up in organs like the heart and kidneys, leading to their failure. The study will use a combination of treatments known as D-VCd Therapy, which includes the medications Daratumumab, Cyclophosphamide, Bortezomib, and Dexamethasone. Additionally, a substance called Sargramostim will be used. Sargramostim is known to help the body produce more white blood cells and may assist in breaking down amyloid deposits.

The purpose of this study is to evaluate how effective and safe this combination of treatments is for patients who have not yet received treatment for Light Chain Amyloidosis. Participants in the study will receive these medications and be monitored for their response to the treatment and any side effects they may experience. The study aims to see if the treatment can lead to a complete response in the blood and improve the function of affected organs.

The study will take place over a period of time, during which participants will receive the treatment and undergo regular check-ups to assess their health and the progress of the disease. The goal is to determine if this combination of treatments can provide a better outcome for patients with Light Chain Amyloidosis by reducing the amyloid deposits and improving organ function.

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 assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a series of tests to ensure the patient meets the study criteria.
Step 2
Treatment initiation
The treatment begins with the administration of a combination therapy known as D-VCd, which includes the medications daratumumab, cyclophosphamide, bortezomib, and dexamethasone.
Daratumumab is given as a subcutaneous injection at a dose of 1800 mg.
Cyclophosphamide is administered orally in the form of coated tablets.
Bortezomib is provided as a subcutaneous injection.
Dexamethasone is taken orally in tablet form at a dose of 20 mg.
Step 3
Ongoing treatment
The treatment regimen is repeated in cycles. Each cycle lasts for a specified period, during which the medications are administered according to the study protocol.
Regular monitoring is conducted to assess the patient's response to the treatment and to manage any side effects.
Step 4
Additional medication
In addition to the D-VCd therapy, sargramostim is used to support the treatment. Sargramostim is a medication that helps stimulate the production of white blood cells and is administered as an injection.
Step 5
Evaluation and follow-up
Throughout the study, evaluations are conducted to measure the effectiveness of the treatment. This includes assessing the percentage of patients who achieve a complete hematologic response.
Safety assessments are also performed to monitor for any serious adverse events.
Step 6
Completion of study
The study is expected to conclude by September 2026. At the end of the study, a final assessment is conducted to evaluate the overall outcomes and the patient's health status.

Who can join the trial?

10 criteria

You must be **18 years of age or older**.
You or your legal representative must sign an **informed consent form**. This means you understand the study and agree to participate.
You need a **diagnosis of AL amyloidosis**. This is confirmed by finding specific materials in tissue samples using special tests.
You must have **measurable disease** of AL amyloidosis. This means certain levels of proteins in your blood are present, which can be measured.
One or more of your organs must be affected by AL amyloidosis.
Your **performance status** should be 0, 1, or 2 on the **ECOG scale**. This scale measures how well you can perform daily activities.
Your **blood test results** must meet specific criteria, such as having enough white blood cells, red blood cells, and platelets.
If you are a woman who can have children, you must have a **negative pregnancy test** and agree to use **reliable birth control** during the study and for a certain period after.
If you are a woman, you must agree not to **donate eggs** during the study and for a certain period after.
If you are a man, you must agree to use **birth control** if you are sexually active with a woman who can have children. You must also agree not to **donate sperm** during the study and for a certain period after.

Who cannot join the trial?

10 criteria

Patients who have a different type of amyloidosis other than AL amyloidosis. AL amyloidosis is a condition where abnormal proteins called amyloid fibrils build up in organs like the heart or kidneys.
Patients who have received any other treatment for AL amyloidosis within a certain period before the study starts.
Patients with severe heart problems that are not stable or controlled.
Patients with severe kidney problems that are not stable or controlled.
Patients with active infections that require treatment.
Patients who have had another type of cancer within the last five years, except for certain skin cancers or other specific conditions.
Patients who are pregnant or breastfeeding.
Patients who have a known allergy to any of the study medications or their ingredients.
Patients who have participated in another clinical trial within a certain period before this study.
Patients with any other medical condition that the study doctors believe would make it unsafe for them to participate.

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Verified sites

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

Verified
Narodowy Instytut Onkologii Im. Marii Sklodowskiej-Curie-Panstwowy Instytut Badawczy
Cracow, Poland
Verified
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
Responsive
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Warsaw, Poland

Trial locations

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Investigated drugs

Sargramostim
is a medication used to stimulate the production of white blood cells. It helps the body fight infections and recover from treatments that can lower white blood cell counts.
Daratumumab
is a medication used to treat certain types of blood disorders. It works by targeting and destroying specific cells in the immune system that are involved in the disease process.
Cyclophosphamide
is a chemotherapy drug that helps to slow or stop the growth of cancer cells. It is used in combination with other medications to treat various types of cancer and blood disorders.
Bortezomib
is a medication that interferes with the growth of cancer cells and slows their spread in the body. It is used to treat certain types of blood cancers.
Dexamethasone
is a steroid that helps reduce inflammation and suppresses the immune system. It is often used in combination with other medications to treat a variety of conditions, including certain types of cancer and blood disorders.

What is already known about the treatment

Daratumumab
This medication is administered intravenously and is part of the D-VCD regimen used in the treatment of light chain amyloidosis. It is currently recognized in medical literature as an effective therapy when combined with other drugs like bortezomib, cyclophosphamide, and dexamethasone. The main therapeutic indication is to target and destroy clonal plasma cells, which are responsible for the production of harmful immunoglobulin light chains. At the molecular level, daratumumab works by binding to the CD38 protein on the surface of these cells, leading to their destruction. It is classified as a monoclonal antibody.
Sargramostim
This medication is administered via injection and is being studied in combination with the D-VCD regimen for light chain amyloidosis. It is currently used in the USA for mobilizing progenitor cells and supporting white blood cell reconstruction after bone marrow transplantation, among other uses. The main therapeutic indication in this context is to enhance the phagocytosis of amyloid deposits by activating tissue phagocytes. At the molecular level, sargramostim stimulates the production and function of white blood cells, including macrophages and neutrophils, which can help clear amyloid deposits. It is classified as a recombinant granulocyte macrophage colony-stimulating factor (GM-CSF).

Investigated diseases

Light Chain Amyloidosis – This disease is a type of plasma cell disorder where misfolded immunoglobulin light chains form amyloid fibrils. These fibrils deposit in various organs, most commonly the heart and kidneys, leading to their dysfunction. The accumulation of these deposits can cause significant organ damage over time. The disease is part of a larger group of conditions known as amyloidoses, which are characterized by abnormal protein deposits. The progression involves the continuous buildup of amyloid fibrils, which can eventually lead to organ failure if not managed.