Active not recruitingPHASE3INTERVENTIONAL

Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis

This research study is investigating a new treatment approach for myelofibrosis, a type of blood cancer where the bone marrow doesn't work properly. The study combines two existing medications, Selinexor and Ruxolitinib, to see if working together they can be more effective and safe for patients who haven't yet received a common type of myelofibrosis treatment called a JAK inhibitor. The study has two main parts: an earlier part that looked at safe doses, and an ongoing part that compares the new combination against Ruxolitinib alone (with a 'dummy' pill instead of Selinexor) to find out if it truly helps improve the condition.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Karyopharm Therapeutics Inc

Enrolment target

353

Start

11 Mar 2021

Estimated completion

01 Mar 2028

Myelofibrosis

What is this study about?

This study is exploring a new way to treat myelofibrosis, a condition affecting your bone marrow where it doesn't make enough healthy blood cells. This can lead to tiredness, a swollen spleen, and other symptoms. The study is particularly for people who haven't yet received a specific type of treatment called a 'JAK inhibitor', which is a common medication for myelofibrosis.

The researchers want to see if combining two drugs, Selinexor and Ruxolitinib, is better than Ruxolitinib on its own. Ruxolitinib is a drug that helps manage swelling of the spleen and other symptoms in myelofibrosis. Selinexor works in a different way, and the idea is that using them together might offer a more powerful or effective treatment for the condition. The study aims to confirm if this combination is safe and helps improve patients' myelofibrosis symptoms and overall health.

This kind of research is really important because it helps doctors find better ways to treat myelofibrosis and improve the lives of people living with it. By participating in a study like this, you could be contributing to new medical knowledge that benefits many others in the future.

Key takeaways

This study is testing a new combination treatment for myelofibrosis.
It's for people who haven't had previous JAK inhibitor treatment.
The study compares Selinexor + Ruxolitinib against Ruxolitinib + a dummy pill.
Participation involves taking study medication and attending regular health checks.
It's a 'double-blind' study, so you won't know which treatment you receive.
You can stop participating at any time if you wish.

Who may be eligible?

To be considered for this study, you would generally need to be at least 18 years old and have received a diagnosis of myelofibrosis. This could be primary myelofibrosis or myelofibrosis that developed after other blood conditions like essential thrombocythemia or polycythemia vera.

You should also be experiencing active symptoms of myelofibrosis, such as a swollen spleen, and your condition needs to have a certain level of risk, as determined by doctors. It's important that you haven't previously been treated with a JAK inhibitor or a drug called Selinexor or similar medicines called XPO1 inhibitors. Your general health and ability to carry out daily activities will also be assessed.

There are also some reasons why you might not be able to join. For example, if you have very high levels of immature blood cells (called 'blasts') in your blood or bone marrow, or if you've already had certain treatments for myelofibrosis, you wouldn't be eligible for this particular study.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with myelofibrosis?
Are you currently experiencing symptoms from your myelofibrosis?
Have you NOT previously taken a JAK inhibitor treatment for myelofibrosis?
Are you able to manage your daily activities without too much difficulty?

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 would first go through a screening process to check if you meet all the requirements. If you're eligible, you would then be given either the Selinexor and Ruxolitinib combination, or Ruxolitinib along with a dummy pill (placebo) instead of Selinexor. This is a "double-blind" study, meaning neither you nor your doctor would know which combination you are receiving, to ensure the results are fair.

You would need to take your study medication regularly as instructed and attend regular clinic visits. These visits will involve various tests and checks to monitor your health, how you are responding to the treatment, and to see if you have any side effects. These could include blood tests, physical examinations, and possibly scans to check your spleen size. The total duration of your participation would depend on how your treatment is going and the study's protocol, but it would involve ongoing visits and assessments over time.

Potential risks and benefits

Taking part in a clinical trial might offer potential benefits, such as access to a new treatment combination that could improve your symptoms or condition. However, there are also potential risks involved. The study drugs may cause side effects, some of which could be serious. The research team will carefully monitor your health throughout the study and explain all known or potential risks to you. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (166)

UAB Division of Hematology/Oncology
Birmingham, United States
UCLA - Satellite Site
Beverly Hills, United States
City of Hope
Duarte, United States
UCLA - Satellite Site
Encino, United States
City of Hope - Irvine Lennar - Satellite
Irvine, United States
UCLA
Los Angles, United States
The Oncology Institute of Hope & Innovation
Pasadena, United States
USOR - Rocky Mountain Cancer Centers - Aurora
Aurora, United States
Smilow Cancer Hospital - New Haven
New Haven, United States
Georgetown Lombardi Comprehensive Center
Washington D.C., United States
Norton Cancer Institute - Saint Matthews
Louisville, United States
Maryland Oncology Hematology-Satellite
Annapolis, United States

+154 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. This can lead to too few healthy blood cells, a swollen spleen, and other symptoms.

What are Selinexor and Ruxolitinib?

These are two different medications. Ruxolitinib is a common drug used to treat myelofibrosis. Selinexor is another drug that works in a different way, and this study is exploring using them together.

What does 'JAK inhibitor (JAKi) treatment-naïve' mean?

It means you have not had a type of myelofibrosis treatment called a JAK inhibitor before. This study is specifically for people new to this kind of treatment.

What is a 'double-blind' study?

In a double-blind study, neither you nor your doctor knows if you are receiving the active study drug or a placebo (dummy pill). This helps ensure the study results are unbiased and reliable.

Can I leave the study if I change my mind?

Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.