RecruitingRare disease

Study on Venetoclax and Rituximab for Patients with Waldenström’s Macroglobulinemia

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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 the effectiveness of a treatment for Waldenström’s Macroglobulinemia, a rare type of cancer that affects white blood cells. The study will explore the use of a medication called Venetoclax, also known by its code name ABT-199, in combination with another medication called Rituximab. The goal is to compare this combination to a different treatment regimen that includes Dexamethasone, Rituximab, and Cyclophosphamide.

The purpose of the study is to determine how well the combination of Venetoclax and Rituximab works compared to the other treatment option in patients who have not been treated for Waldenström’s Macroglobulinemia before. Participants in the study will receive either the Venetoclax and Rituximab combination or the Dexamethasone, Rituximab, and Cyclophosphamide combination. The study will last for several months, during which time participants will receive their assigned treatment and be monitored for their response to the therapy.

Throughout the study, researchers will assess the participants' health and the effectiveness of the treatments. The main focus will be on the rate of complete or very good partial remission, which means the cancer has significantly decreased or disappeared. The study will also look at other outcomes, such as the time it takes for the treatment to start working, how long the response lasts, and the overall survival of the participants. Safety and quality of life will also be important considerations during the trial.

The research process

The trial runs in 7 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 the diagnosis of Waldenström’s Macroglobulinemia. This includes a review of medical history and laboratory tests to ensure eligibility.
Pathological specimens are sent to a central reference center to determine specific genetic mutations if not previously identified.
Step 2
Treatment group assignment
Participants are randomly assigned to one of two treatment groups. One group receives a combination of venetoclax and rituximab, while the other group receives dexamethasone, rituximab, and cyclophosphamide.
Step 3
Medication administration
For the venetoclax and rituximab group, venetoclax is taken orally as a film-coated tablet. Rituximab is administered through an infusion.
For the dexamethasone, rituximab, and cyclophosphamide group, dexamethasone is taken orally, rituximab is given as an intravenous infusion, and cyclophosphamide is taken orally.
Step 4
Treatment duration
The treatment is administered over a specified period, with regular monitoring to assess the response and any side effects. The exact duration and frequency of medication administration are determined by the study protocol.
Step 5
Follow-up assessments
Regular follow-up assessments are conducted to evaluate the effectiveness of the treatment. This includes monitoring for complete or partial remission and any changes in symptoms.
Assessments may include physical examinations, blood tests, and imaging studies.
Step 6
End of treatment evaluation
At the end of the treatment period, a comprehensive evaluation is performed to determine the overall response to the therapy.
The evaluation includes measuring the rate of complete or very good partial remission 12 months after starting the treatment.
Step 7
Long-term follow-up
Participants are monitored for long-term outcomes such as progression-free survival, overall survival, and quality of life.
The study aims to compare the response rates between different genetic profiles of the disease.

Who can join the trial?

13 criteria

Must have a confirmed diagnosis of **Waldenström’s Macroglobulinemia (WM)**, a type of blood cancer, as per specific guidelines. Diagnosis must be confirmed by a central reference center.
Women who can have children must have a negative pregnancy test and agree to use effective birth control during the study and for 12 months after.
Men must agree not to father a child during the study and for 12 months after, and advise their partners to use effective birth control. They must also refrain from donating sperm during this time.
Must voluntarily sign a consent form indicating understanding and willingness to participate in the study.
Must be part of a social security scheme (only relevant for participants in France).
Must have **de novo WM**, meaning newly diagnosed and in need of treatment.
Must have at least one symptom or condition related to WM, such as fever, night sweats, weight loss, fatigue, or other specific symptoms like organ enlargement or blood-related issues.
Must be 18 years of age or older.
Must have a life expectancy of more than 3 months.
Must have a performance status of 2 or less, which means being able to carry out all self-care but unable to carry out any work activities.
Must have a baseline platelet count of at least 50x10^9/L and a neutrophil count of at least 0.75x10^9/L, unless affected by the disease.
Must have adequate liver function, with specific enzyme levels within acceptable limits.
Must have adequate kidney function, with a creatinine clearance of at least 30 mL/min, which measures how well the kidneys are working.

Who cannot join the trial?

9 criteria

Patients who have a different type of cancer other than Waldenström’s Macroglobulinemia.
Patients who are not within the specified age range for the study.
Patients who are not able to follow the study procedures or take the study medications as required.
Patients who have a medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial recently.
Patients who have an allergy or severe reaction to any of the study medications.
Patients who have a serious infection or illness that could interfere with the study.
Patients who have a history of drug or alcohol abuse that could affect their ability to participate in the study.

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Verified sites

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

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Alexandra Hospital
Athens, Greece
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Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz
Mainz, Germany
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Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH
Ulm, Germany

Trial locations

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Investigated drugs

Venetoclax
is a medication used in this trial to help treat Waldenström’s Macroglobulinemia. It works by targeting and blocking a specific protein in cancer cells, which can help to stop the growth of these cells and potentially lead to their death.
Rituximab
is a medication that is used in combination with other drugs in this trial. It is a type of therapy that targets specific cells in the immune system, helping to destroy cancer cells and slow down the progression of the disease.
Dexamethasone
is a medication used in this trial as part of a combination therapy. It is a type of steroid that helps reduce inflammation and can also help kill cancer cells or make them more sensitive to other cancer treatments.
Cyclophosphamide
is a chemotherapy drug used in this trial. It works by interfering with the DNA of cancer cells, which can prevent them from growing and dividing, ultimately leading to their destruction.

What is already known about the treatment

Venetoclax
Venetoclax is administered orally in the form of tablets. It is currently being studied in combination with Rituximab for its effectiveness in treating Waldenström’s Macroglobulinemia, a type of blood cancer. The medication works by inhibiting a protein called BCL-2, which helps cancer cells survive, thereby promoting their death. Venetoclax is classified as a BCL-2 inhibitor and is part of targeted cancer therapies.
Rituximab
Rituximab is given through intravenous infusion. It is widely used in medical practice and literature for treating various types of blood cancers, including Waldenström’s Macroglobulinemia. The drug targets the CD20 protein found on the surface of certain blood cells, leading to their destruction. Rituximab is classified as a monoclonal antibody and is a key component in cancer immunotherapy.

Investigated diseases

Waldenström’s Macroglobulinemia – This is a rare type of non-Hodgkin lymphoma characterized by an overproduction of a specific type of antibody called IgM. The disease originates from white blood cells known as B lymphocytes, which become cancerous and accumulate in the bone marrow. As these cells multiply, they can interfere with normal blood cell production, leading to symptoms such as fatigue, bleeding, and increased risk of infections. Over time, the excess IgM can cause the blood to thicken, potentially leading to complications like vision problems and neurological issues. The progression of the disease can vary, with some individuals experiencing slow development of symptoms, while others may have more rapid changes.