Active not recruitingPHASE2INTERVENTIONAL

Efficacy and Safety of Carfilzomib in Combination With Ibrutinib vs Ibrutinib in Waldenström's Macroglobulinemia

This research trial is investigating a new way to treat Waldenström's Macroglobulinemia (WM), a type of blood cancer. We're comparing two treatment approaches: one group will receive a combination of Carfilzomib and Ibrutinib, while another group will receive Ibrutinib on its own. Previous studies have shown that Ibrutinib is a good treatment, but it doesn't always lead to the best possible outcomes for all patients, especially those with certain genetic patterns in their cancer cells. Carfilzomib has shown promise in helping these specific patient groups. By combining these two drugs, we hope to achieve better and longer-lasting responses, particularly for patients with a specific genetic change called the CXCR4 mutation. This Phase 2 study aims to see if this combination is more effective and well-tolerated.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Christian Buske

Enrolment target

Start

18 Aug 2021

Estimated completion

01 May 2028

Waldenstrom Macroglobulinemia

What is this study about?

This study is about finding better ways to treat a type of blood cancer called Waldenström's Macroglobulinemia, or WM for short. Currently, standard chemotherapy treatments for WM don't always fully clear the cancer, and the effects often don't last very long. This means doctors are always looking for new and more effective ways to help patients. Our aim is to explore 'chemotherapy-free' options, which can be particularly good for patients, as they often have fewer side effects than traditional chemotherapy.

One drug called Ibrutinib has already been approved in the UK for treating WM because it works well and causes fewer side effects than chemotherapy. However, while Ibrutinib is good, it doesn't always lead to a complete disappearance of the cancer, and it might not work as well for everyone, especially for those whose cancer cells have certain genetic differences. For example, if your WM has a specific change called a CXCR4 mutation, Ibrutinib alone might not be as effective. Another drug, Carfilzomib, has shown encouraging results in previous studies, particularly for patients with these specific genetic changes.

So, in this study, we want to see if combining Carfilzomib with Ibrutinib can improve the treatment outcomes for people with WM. We especially want to find out if this combination works better for patients with the CXCR4 mutation. We also want to check if this combination is effective for patients whose cancer cells have another genetic type called MYD88 wildtype. Ultimately, we hope this combination will lead to more patients having their cancer go into 'very good partial response' or 'complete remission', meaning the cancer cells are greatly reduced or completely gone. This Phase 2 study will carefully assess how well this new combination works and how safe it is.

Key takeaways

This study is testing if combining Carfilzomib and Ibrutinib is better for WM than Ibrutinib alone.
It aims to achieve stronger and longer-lasting responses, especially for patients with certain genetic features.
Participation involves regular clinic visits, blood tests, and close monitoring.
This is a Phase 2 trial, focusing on treatment effectiveness and safety.
Both newly diagnosed and relapsed/refractory patients with WM may be eligible.
You can withdraw from the study at any time without affecting your ongoing care.

Who may be eligible?

To be considered for this study, you would first need to have a confirmed diagnosis of Waldenström's Macroglobulinemia (WM). This would be based on medical tests and a review of your tissue samples by specialists. These tests would also check the genetic makeup of your WM cells, specifically for changes in key genes like MYD88 and CXCR4, as this study is particularly interested in how treatments work for different genetic types.

Beyond a confirmed diagnosis, you'd need to be experiencing certain symptoms or signs of your WM that indicate treatment is needed. These could include general symptoms like fever, night sweats, weight loss, or feeling very tired, or specific issues like an enlarged spleen or liver, nerve problems, or changes in your blood count such as low haemoglobin or platelets. Your doctors will explain these in more detail.

This study is open to both people who have been newly diagnosed with WM and those whose WM has returned or hasn't responded to previous treatments. You must be at least 18 years old, but there's no upper age limit. Your medical team will carefully review all your health information to ensure this study is a safe and suitable option for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have a confirmed diagnosis of Waldenström's Macroglobulinemia (WM)?
Am I experiencing symptoms or issues related to my WM that need treatment?
Am I at least 18 years old?
Has my doctor reviewed the genetic information of my WM cells (MYD88 and CXCR4 status)?
Am I willing and able to attend regular clinic visits and follow the study schedule?
Have I discussed all my current medications and health conditions with the study team?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be assigned to one of two groups: one receiving the combination of Carfilzomib and Ibrutinib, and the other receiving Ibrutinib on its own. The study will involve regular visits to the clinic for assessments, which will include blood tests, physical examinations, and possibly other scans or procedures to monitor your health and how well the treatment is working. Medication will be provided to you as part of the study. The total duration of your participation will depend on your individual response to treatment and the study plan, but it will involve ongoing treatment and follow-up appointments for a period that your doctor will discuss with you. You'll be closely monitored throughout the study.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and risks. A potential benefit of this study is that the combination treatment might be more effective than current standard treatments, potentially leading to better control of your WM or improved responses. However, there's no guarantee this treatment will work for everyone, and it might not be better than existing options. As with all medications, both Carfilzomib and Ibrutinib can cause side effects. The research team will discuss all known potential risks and side effects with you in detail, and you'll be closely monitored for any issues throughout the study. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without affecting your future medical care.

Locations (17)

Uniklinikum Salzburg
Verified postcode
Salzburg, Austria
Medizinische Universität Wien
Verified postcode
Vienna, Austria
Vivantes Klinikum am Urban
Verified postcode
Berlin, Germany
Ev. Diakoniekrankenhaus
Verified postcode
Bremen, Germany
Kath. St.-Johannes-Gesellschaft Dortmund gGmbH
Verified postcode
Dortmund, Germany
Gemeinschaftspraxis Mohm / Prange-Krex
Verified postcode
Dresden, Germany
OncoResearch Lerchenfeld GmbH
Verified postcode
Hamburg, Germany
MediProject Onkologisches Ambulanzzentrum Hannover
Verified postcode
Hanover, Germany
Praxis für Hämatologie und Onkologie, onkologische Tagesklinik-VK&K Studien GbR
Verified postcode
Landshut, Germany
Praxis Dr. Vehling-Kaiser
Verified postcode
Landshut, Germany
Hämatologie/Onkologie München Pasing MVZ GmbH
Verified postcode
Munich, Germany
Kliniken Ostalb, Staufenklinikum Schw. Gmuend
Verified postcode
Mutlangen, Germany

Common questions

What is Waldenström's Macroglobulinemia (WM)?

WM is a rare type of slow-growing blood cancer that affects white blood cells called lymphocytes, leading to the production of abnormal proteins in the blood.

What are Carfilzomib and Ibrutinib?

These are prescription medicines used to treat certain blood cancers. Ibrutinib is already approved for WM, and Carfilzomib is being studied to see if it can improve how well Ibrutinib works.

Why is this study looking at genetics?

The study examines specific genetic changes in your WM cells (like MYD88 and CXCR4) because they can affect how well certain treatments, including Ibrutinib, work. This helps us understand which treatments are best for different patients.

Will I know which treatment I'm getting?

In some studies, you might not know which specific treatment you're receiving (combination or Ibrutinib alone) until the end of the trial. Your doctor will explain this process, called 'blinding'.

What does 'Phase 2' mean for a clinical trial?

Phase 2 trials further test the safety of a new treatment and start to gather information on how well it works for a specific condition. It's a step before larger trials that compare it to standard treatments.