Active not recruitingPHASE2INTERVENTIONAL

An Open-label, Phase 2 Study of ACP-196 in Subjects With Waldenström Macroglobulinemia

This study is looking at a new medicine called acalabrutinib to treat Waldenström Macroglobulinemia (WM), which is a rare type of blood cancer. Researchers want to find out if acalabrutinib is safe, how it affects the body, and how well it works in people with WM. WM causes the body to make too much of a certain protein and can lead to problems. Current treatments exist, but scientists are always looking for new and better options. Acalabrutinib is a type of drug that targets a specific pathway in cancer cells, similar to another approved medicine, but it's new and needs careful testing. This study will help us understand if it could be a helpful treatment for WM.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Acerta Pharma BV

Enrolment target

107

Start

11 Sep 2014

Estimated completion

31 Dec 2026

Waldenström Macroglobulinemia (WM)

What is this study about?

Waldenström Macroglobulinemia (WM) is a rare type of cancer that affects certain white blood cells. These cells produce a protein called IgM, and in WM, too much IgM is made. This can cause the blood to become thick and lead to various health problems. While there are treatments available for WM, scientists are always working to find new and more effective options, or ones with fewer side effects.

This study is focusing on a new medicine called acalabrutinib. This drug works by blocking a specific signal inside the cancer cells, which helps stop them from growing and multiplying. You might have heard of a similar medication called ibrutinib, which is already used to treat WM and other similar conditions. Acalabrutinib is designed to work in a similar way but is a newer version that shows promise in early tests.

The main goals of this study are to carefully check how safe acalabrutinib is for people with WM. Researchers also want to understand how the body handles the medicine – how it's absorbed, used, and cleared from the body. Finally, and very importantly, they want to see if acalabrutinib is effective at treating WM and helping patients feel better or slow down the disease’s progress. This type of study (called a Phase 2 study) is a crucial step in bringing new medicines to patients.

Key takeaways

The study is testing a new medicine, acalabrutinib, for Waldenström Macroglobulinemia (WM).
It aims to check the safety, how the body handles it, and if it helps treat WM.
Participation requires regular clinic visits and health checks.
It's for people with WM who have either been previously treated or are newly diagnosed but cannot have standard chemotherapy.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to be at least 18 years old. The study is open to both men and women. You must have a confirmed diagnosis of Waldenström Macroglobulinemia (WM).

If you've already had treatment for WM, you can join if your WM has come back or hasn't responded well to at least one previous treatment. If you haven't been treated for WM before, you can join if you need treatment and either don't want to receive standard chemotherapy combined with immunotherapy, or if other health issues mean you can't have those standard treatments. Your WM also needs to show certain signs, like a high level of a protein called IgM in your blood, or specific sized swollen lymph nodes.

There are also some general health requirements. For example, you need to be well enough to take part in all the study activities. There are other reasons why you might not be able to join, such as having certain other serious medical conditions or recent cancers (other than some common skin cancers or very early-stage cervical cancer). The medical team will check all your health details carefully to make sure the study is safe and right 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.

Are you 18 years old or older?
Do you have a confirmed diagnosis of Waldenström Macroglobulinemia?
If you've had WM treatment before, has your WM come back or not responded well?
If you've never been treated for WM, are you unable to have standard chemotherapy or prefer not to?
Are you able to swallow capsules without difficulty?
Do you feel well enough to attend regular clinic visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given the study medicine, acalabrutinib, which you will take by mouth as capsules. The study will involve regular visits to the clinic so the medical team can monitor your health and how you're responding to the treatment. These visits will include various assessments like blood tests to check how the medicine is working and for any side effects, as well as general health check-ups.

You will need to be able to swallow capsules without difficulty. During the study, it's very important for women who can get pregnant to use highly effective contraception, and to continue doing so for 2 days after your last dose of the study medicine. The total duration of your participation, including how long you take the medicine and any follow-up visits, will be explained in detail by the study team. They will also discuss all the specific procedures and the time commitment involved.

Potential risks and benefits

Taking part in any clinical study has potential benefits and risks. You might benefit from receiving a new treatment that could help manage your Waldenström Macroglobulinemia if existing therapies haven't worked well or if you can't have standard treatments. However, it's important to remember that this is a new medicine, and its full effects are still being studied. There's no guarantee it will work for everyone, and it might not work for you. Potential risks include side effects from the medicine, which will be carefully monitored by the study team. These could range from mild to more serious, and the team will discuss all known possible side effects with you before you agree to participate. You will also have many tests and procedures as part of the study. It is very important to remember that you can choose to leave the study at any time, for any reason, without it affecting your medical care.

Locations (38)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Research Site
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Santa Barbara, United States
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Aurora, United States
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Washington D.C., United States
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Minneapolis, United States
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New York, United States
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Nashville, United States
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Austin, United States
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Bedford, United States
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Houston, United States
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Vancouver, United States
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Aurillac, France
Research Site
Unverified
Clermond Ferrand, France

Common questions

What is Waldenström Macroglobulinemia (WM)?

It's a rare type of blood cancer where your body makes too much of a specific protein, which can cause health issues.

What is acalabrutinib?

It's a new medicine being tested. It works by blocking a signal in cancer cells to stop them from growing.

Is acalabrutinib already approved for WM?

No, it's still being studied in clinical trials like this one to see if it's safe and effective for WM.

Will I get regular checks if I join?

Yes, you'll have regular visits, blood tests, and health checks to monitor your progress and safety.

Can I leave the study if I change my mind?

Absolutely, you can withdraw from the study at any time, and it won't affect your regular medical care.