RecruitingPHASE3INTERVENTIONAL

Study of Navtemadlin add-on to Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib

Researchers are investigating if adding an experimental drug called navtemadlin to ruxolitinib can further help people with myelofibrosis. Myelofibrosis is a condition where the bone marrow, which makes blood cells, becomes scarred, leading to problems like tiredness and an enlarged spleen. This study is for people who are already taking ruxolitinib but still have symptoms or signs that their condition isn't as well-controlled as hoped. Participants will first take ruxolitinib as usual. If their condition isn't optimally controlled, they will then be randomly assigned to either receive navtemadlin or a dummy drug (placebo) alongside their ruxolitinib. Neither the participants nor their doctors will know which add-on treatment they are receiving, so researchers can fairly compare the effects.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Kartos Therapeutics, Inc.

Enrolment target

600

Start

03 Jun 2024

Estimated completion

31 Dec 2028

Myelofibrosis Post-PV MF Post-ET Myelofibrosis Primary Myelofibrosis MF

What is this study about?

This clinical trial is designed to investigate a new treatment approach for myelofibrosis, a rare blood cancer that affects the bone marrow. The current standard treatment for many people with myelofibrosis is a medication called ruxolitinib. However, for some individuals, ruxolitinib alone may not fully control their symptoms or stop the disease from progressing as much as needed. This study is exploring whether adding a new drug, navtemadlin, to ruxolitinib can offer additional benefits for these patients.

The study starts with everyone receiving ruxolitinib. Doctors will then check how well each patient is responding to ruxolitinib alone. If a patient’s condition isn’t responding as well as hoped, they will be invited to continue into the next phase of the study. In this phase, they will be put into one of two groups by chance – like flipping a coin. One group will receive navtemadlin alongside their ruxolitinib, and the other group will receive a placebo (a dummy drug that looks like navtemadlin but has no active ingredient) along with their ruxolitinib. This is done to fairly compare if navtemadlin truly makes a difference.

Neither the patients nor their doctors will know which additional treatment (navtemadlin or placebo) is being given. This is called a 'blinded' study and helps ensure that the results are as unbiased as possible. The main goal is to see if adding navtemadlin improves how people with myelofibrosis respond to treatment compared to just continuing with ruxolitinib alone.

Key takeaways

This study evaluates adding a new drug, navtemadlin, to ruxolitinib for myelofibrosis.
It's for people whose myelofibrosis isn't fully controlled by ruxolitinib alone.
Participation involves taking ruxolitinib first, then potentially receiving navtemadlin or a placebo.
The study is 'blinded', meaning you won't know if you're getting the active drug or a dummy pill.
Regular monitoring and clinic visits will be part of the study.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you must first have been diagnosed with a specific type of myelofibrosis, such as primary myelofibrosis or myelofibrosis that developed after essential thrombocythaemia or polycythaemia vera. Your general health and ability to perform daily activities will be assessed, with a good overall physical condition generally required. Importantly, you must not have previously taken any medications known as JAK inhibitors (like ruxolitinib) before starting this trial.

There are also reasons why you might not be able to join the study. For example, if you've had your spleen removed or have had radiation therapy to your spleen recently. You also can't have received certain other specific treatments for your myelofibrosis in the past. If you have a very high number of immature blood cells (blasts) in your blood or bone marrow, or if you are eligible for a bone marrow transplant, you might also not be able to participate.

If you meet these initial criteria and start taking ruxolitinib within the study, you'll then need to show that your myelofibrosis isn’t fully controlled by ruxolitinib alone to continue with the navtemadlin or placebo part of the study. At this point, your general health physical assessment will be re-evaluated and your blast count will also be checked again.

Quick self-check

Do I have a confirmed diagnosis of myelofibrosis (primary, post-PV, or post-ET)?
Am I generally in good health and able to perform daily activities?
Have I never taken a JAK inhibitor medication before enrolling in this study?
Have I not had my spleen removed recently or received spleen radiation in the last 3 months?
Do I currently have fewer than 10% immature blood cells (blasts) in my blood or bone marrow?

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 first receive ruxolitinib treatment (if you are not already taking it). Your doctors will monitor your response to this treatment over a period. If your myelofibrosis isn't controlled as well as hoped, you may then be asked to continue into the next stage.

In the second stage, you'll be assigned by chance to receive either navtemadlin or a placebo (a dummy drug) in addition to your ongoing ruxolitinib. Both you and your doctors will not know which one you are receiving. You will need to take these medications regularly as instructed. Throughout the study, you will have regular clinic visits, blood tests, and other medical examinations to check your health, how you are responding to the treatment, and to monitor for any side effects. The exact number of visits and tests will be explained by the study team, along with the total duration of your participation in the study.

Potential risks and benefits

Taking part in this study might offer a potential benefit if navtemadlin proves to be more effective than ruxolitinib alone in controlling your myelofibrosis. However, as with any medication, there's always a possibility of experiencing side effects, including those that are currently unknown, or that the treatment may not improve your condition. The study team will carefully monitor your health throughout. You have the full right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (215)

UAB Hospital
Birmingham, United States· Recruiting
Banner MD Anderson Cancer Center
Gilbert, United States· Recruiting
Mayo Clinic - Phoenix
Phoenix, United States· Recruiting
UC San Diego Moores Cancer Center
La Jolla, United States· Recruiting
UCLA Hematology/Oncology Clinic - Los Angeles
Los Angeles, United States· Recruiting
Scripps Health, Prebys Cancer Center
San Diego, United States· Recruiting
The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance, United States· Recruiting
Rocky Mountain Cancer Centers - Aurora
Aurora, United States· Recruiting
Medical Oncology Hematology Consultants, PA
Newark, United States· Recruiting
MedStar Georgetown University Hospital, Lombardi Comprehensive Cancer Center
Washington D.C., United States· Recruiting
Mayo Clinic - Jacksonville
Jacksonville, United States· Recruiting
University of Miami
Miami, United States· Recruiting

+203 more sites — see the official record for the full list.

Common questions

What is myelofibrosis?

Myelofibrosis is a condition where the bone marrow, which makes blood cells, becomes scarred, leading to problems like tiredness and an enlarged spleen.

What is ruxolitinib?

Ruxolitinib is a medication that is commonly used to treat myelofibrosis.

What does 'suboptimal response' mean?

It means your myelofibrosis isn't as well-controlled as doctors hope, even though you're taking ruxolitinib.

What is a 'placebo'?

A placebo is a dummy pill that looks like the real medicine but contains no active drug. It helps researchers know if the new drug truly works.

Will I know if I'm getting the new drug or the placebo?

No, neither you nor your doctor will know. This is called a 'blinded' study and helps make the results fair.

How to find out more

John Mei

jmei@kartosthera.com 650-542-0136 Official trial record

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