Active not recruitingPHASE2INTERVENTIONAL

A Study to Evaluate Single Agent Selinexor Versus Physician's Choice in Participants With Previously Treated Myelofibrosis

This study is for people with myelofibrosis, a type of bone marrow disorder, who have already been treated with a common medication called a JAK inhibitor for at least six months, but it hasn't fully controlled their symptoms. The study aims to find out if a new drug called selinexor is a safe and effective treatment option. Participants will be randomly assigned to either receive selinexor or a treatment chosen by their doctor. Doctors will carefully monitor everyone involved to see how well the treatments work, especially in terms of spleen size and overall well-being. This is an important step in finding new ways to help people living with myelofibrosis who need more effective treatments.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Karyopharm Therapeutics Inc

Enrolment target

112

Start

17 Mar 2021

Estimated completion

01 Sep 2026

Myelofibrosis

What is this study about?

This research study is looking for new ways to treat myelofibrosis, which is a condition affecting your bone marrow that can lead to problems like an enlarged spleen and anaemia. Many people with myelofibrosis are treated with medications called JAK inhibitors. However, sometimes these treatments don't work as well as hoped, or their effects wear off over time.

This study is focusing on a new drug called selinexor. It's being compared to other standard treatments that your doctor might choose. The main goal is to see if selinexor is safe for people with myelofibrosis and if it works better than other options, particularly in helping to shrink an enlarged spleen, which is a common problem in this condition. The study is especially for those who have already used a JAK inhibitor for at least six months and still have symptoms.

Participants will be divided into two groups by chance: one group will receive selinexor, and the other will get a treatment chosen by their doctor. This type of study, called a 'Phase 2' trial, helps researchers understand more about the drug's effects before it can become a widely available treatment. By taking part, you could contribute to developing new and better treatments for myelofibrosis.

Key takeaways

This study is testing a new drug, selinexor, for myelofibrosis.
It's for people whose current treatment (JAK inhibitors) isn't working well.
Participants will receive either selinexor or a standard treatment chosen by their doctor.
The main goals are to check safety and how well it helps with symptoms like an enlarged spleen.
This research helps find new ways to treat myelofibrosis.
You can withdraw from the study at any time.

Who may be eligible?

You might be able to join this study if you have been diagnosed with myelofibrosis, including if it developed after essential thrombocythemia or polycythemia vera. You must have already been on a JAK inhibitor treatment for at least six months, and this treatment isn't working as well as it should be, or you can't tolerate it anymore. This might mean your spleen hasn't shrunk enough, has grown bigger, or you developed serious side effects.

You also need to be at least 18 years old. We will check medical details like your blood counts (platelets, white blood cells) and how well your liver and kidneys are working, to make sure it's safe for you to take part. Your general health and ability to carry out daily activities, as assessed by a specific scale (ECOG score of 2 or less), will also be important.

Finally, for safety reasons, we'll need to measure your spleen size using special scans like an MRI or CT scan to confirm it is enlarged enough for the study. If you have hepatitis B or C, there are specific rules about how well it needs to be controlled before you can join.

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 you have a diagnosis of myelofibrosis?
Have you been treated with a JAK inhibitor for at least 6 months?
Is your current JAK inhibitor treatment not working well enough, or are you having difficulties with it?
Are you 18 years old or older?
Have you had a recent scan showing an enlarged spleen?
Are your general health, blood counts, and organ functions generally good enough for study participation?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you will be randomly assigned to one of two groups: either you'll receive the new drug, selinexor, or you'll receive a treatment chosen by your doctor based on what's best for you. This random assignment means neither you nor your doctor can choose which group you are in, like flipping a coin.

Throughout the study, you'll have regular hospital visits for check-ups. These visits will involve blood tests, physical examinations, and scans (like MRI or CT scans) to measure your spleen and assess how well the treatment is working. We will also monitor any side effects you might experience very closely to ensure your safety.

The duration of your participation will depend on how you respond to the treatment and your doctor's assessment. You will continue to receive treatment as long as it's safe and beneficial for you. There will also be follow-up appointments after you stop the study treatment to check on your health.

Potential risks and benefits

Taking part in this study means you could potentially benefit from a new treatment that might help manage your myelofibrosis symptoms, particularly if your current treatment isn't working well. While there are potential benefits, it's also important to be aware of potential risks. Selinexor, like any medication, may have side effects, and these will be carefully explained to you by the study team. You'll be closely monitored for any unwanted effects. Remember, joining a clinical trial is always voluntary. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (21)

The Oncology Institute of Hope and Innovation
Verified postcode
Pasadena, United States
Rocky Mountain Cancer Centers, LLP
Verified postcode
Aurora, United States
Illinois Cancer Specialist
Verified postcode
Niles, United States
Texas Oncology - Northeast Texas
Verified postcode
Tyler, United States
Peking Union Medical College Hospital
Verified postcode
Beijing, China
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Verified postcode
Wuhan, China
Affiliated Hospital of Nantong University
Verified postcode
Nantong, China
The Second Affiliated Hospital of Soochow University
Verified postcode
Suzhou, China
Suzhou University -The First Affiliated Hospital
Verified postcode
Suzhou, China
The First Hospital of Jilin University
Verified postcode
Changchun, China
Sir Run Run Shaw Hospital - Zhejiang University School of Medicine
Verified postcode
Hangzhou, China
Institut de Cancéro-Hématologie
Verified postcode
Brest, France

Common questions

What is myelofibrosis?

Myelofibrosis is a condition where scar tissue builds up in your bone marrow, affecting its ability to make healthy blood cells. This can lead to problems like an enlarged spleen and anaemia.

What is a JAK inhibitor?

JAK inhibitors are a type of medication commonly used to treat myelofibrosis. They work by blocking certain signals in your body that contribute to the disease.

What does 'randomised' mean in a study?

Randomised means that you are put into one of the study groups by chance, like drawing numbers from a hat. This helps ensure the study results are fair and unbiased.

Why is my spleen size important?

An enlarged spleen is a common symptom of myelofibrosis and can cause discomfort. Measuring spleen size helps doctors understand how well treatments are working.

Will I know which treatment I'm getting?

Yes, this is an 'open-label' study, which means both you and your study doctor will know whether you are receiving selinexor or the doctor's choice of treatment.