RecruitingPHASE2INTERVENTIONAL

A Study of Selinexor Monotherapy in Subjects With JAK Inhibitor-naïve Myelofibrosis and Moderate Thrombocytopenia

This research study is investigating a new medication, selinexor, for adults with myelofibrosis. Myelofibrosis is a condition where the bone marrow, responsible for making blood cells, doesn't work properly. This study is specifically for people who haven't previously taken a type of medicine called a JAK inhibitor and have platelet levels that are normal or on the lower side. The main goal is to find out how well selinexor works at reducing the size of the spleen, which can often become enlarged in myelofibrosis. Researchers will also be looking at other effects of the treatment and its safety. The study hopes to offer a new treatment option for this condition.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Karyopharm Therapeutics Inc

Enrolment target

Start

22 Apr 2024

Estimated completion

01 Oct 2028

Myelofibrosis Moderate Thrombocytopenia Mild Thrombocytopenia

What is this study about?

This study is looking into a new medication called selinexor for people diagnosed with a condition known as myelofibrosis. Myelofibrosis is a type of chronic blood cancer where the bone marrow, the spongy tissue inside your bones that makes blood cells, becomes scarred. This scarring disrupts the normal production of blood cells, leading to various symptoms such as fatigue, an enlarged spleen, and low blood counts. The study is specifically for those with myelofibrosis who have not yet received certain other common treatments, known as JAK inhibitors, and who have either normal or slightly low platelet levels. Platelets are tiny blood cells that help your blood clot.

The main purpose of this research is to see how effectively selinexor can reduce the size of an enlarged spleen. An enlarged spleen is a common problem in myelofibrosis and can cause discomfort and other health issues. Researchers will use special scans like MRI or CT to measure spleen size before and during the study. They will also keep a close eye on other effects of the drug, such as how it might improve symptoms like tiredness or night sweats, and importantly, they will monitor its safety to understand any potential side effects.

By taking part, you would be helping doctors and researchers learn more about myelofibrosis and how best to treat it. The hope is that studies like this can lead to new and better treatment options for people living with this condition, helping to manage symptoms and improve quality of life.

Key takeaways

This study is testing a new drug, selinexor, for myelofibrosis.
It's for people who haven't used other specific myelofibrosis drugs (JAK inhibitors).
The main aim is to see if selinexor shrinks an enlarged spleen.
Participation involves regular clinic visits, blood tests, scans, and symptom questionnaires.
You'll be closely monitored for any side effects.
It's an opportunity to access a new treatment and help advance medical knowledge.

Who may be eligible?

To join this study, you would need to have been diagnosed with myelofibrosis, or a similar condition that has become myelofibrosis, based on medical tests. Your spleen would need to be noticeably enlarged, as confirmed by a scan, and you should be experiencing some symptoms related to your myelofibrosis. Your doctors would also assess the severity of your condition, and you would need to fit into certain risk categories.

Your blood test results are important; your platelet count (which helps your blood clot) and neutrophil count (a type of white blood cell) need to be within a certain range. Your liver and kidney health would also be checked to make sure they are working well enough for you to take the study medication. You also shouldn't be eligible for a stem cell transplant at this time.

There are also reasons why you couldn't take part. For instance, if you have a very high number of immature blood cells (called blasts) in your blood or bone marrow, or if you've already had certain other types of treatments for myelofibrosis, particularly a specific group of drugs called JAK inhibitors, or selinexor itself. Women who are pregnant or breastfeeding also cannot participate.

Quick self-check

Have you been diagnosed with myelofibrosis or a related condition?
Do you have an enlarged spleen confirmed by a scan?
Have you *not* taken a JAK inhibitor for your myelofibrosis before?
Are your blood counts (platelets, white blood cells) within acceptable ranges?
Are you able to attend regular clinic visits and complete questionnaires?
Are you currently not pregnant or breastfeeding?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be given one of two doses of the study drug, selinexor, or one of the other established medications (Ruxolitinib, Pacritinib, Momelotinib). Doctors will decide which treatment group you are in. You would take the medication as prescribed by the study team. You would have regular visits to the clinic for medical check-ups, blood tests, and scans, including MRI or CT scans, to measure your spleen size. You will also be asked to complete daily questionnaires about your symptoms to help the researchers understand how you are feeling and if your symptoms are improving. You'll need to provide bone marrow samples at certain times during the study. The total duration of your participation in the study, including follow-up, will be explained to you by the study team.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as access to a new treatment that might help manage your myelofibrosis symptoms and potentially reduce your spleen size before it is widely available. However, there are also potential risks, including side effects from the study medication, which will be discussed in detail by the study team. Your health will be closely monitored 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 it affecting your usual medical care.

Locations (70)

City of Hope - Duarte Main Site
Duarte, United States· Recruiting
Maryland Oncology Hematology - Independent of SCRI/ US Oncology
Columbia, United States· Recruiting
Weill Cornell Medicine NewYork-Presbyterian
New York, United States· Recruiting
Duke University
Durham, United States· Recruiting
Cleveland Clinic
Cleveland, United States· Recruiting
MD Anderson
Houston, United States· Recruiting
Huntsman Cancer Institute
Salt Lake City, United States· Recruiting
UZ Gent
Ghent, Belgium· Recruiting
UZ Leuven - Campus Gasthuisberg
Leuven, Belgium· Recruiting
University Multiprofile Hospital for Active Treatment Sveti George - Base 1
Plovdiv, Bulgaria· Recruiting
University Hospital Sv.Ivan Rilski - Sofia
Sofia, Bulgaria· Recruiting
University Multiprofile Hospital for Active Treatment Aleksandrovska
Sofia, Bulgaria· Recruiting

+58 more sites — see the official record for the full list.

Common questions

What is myelofibrosis?

Myelofibrosis is a condition where your bone marrow, which makes blood cells, becomes scarred, leading to problems with blood cell production and an enlarged spleen.

What are JAK inhibitors?

JAK inhibitors are a type of medication used to treat myelofibrosis. This study is for people who haven't tried them before.

What does 'moderate thrombocytopenia' mean?

It means you have a moderate level of low platelets, which are small cells in your blood that help it clot.

How will my spleen size be measured?

Your spleen size will be measured using imaging scans like an MRI or CT scan.

Will I have to pay to be in the study?

No, you will not have to pay for the study medication or for appointments directly related to the research.

How to find out more

Karyopharm Medical Information

clinicaltrials@karyopharm.com (888) 209-9326 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.