RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of Oral Nuvisertib (TP-3654) in Patients With Myelofibrosis

This research study is looking into a new medication called nuvisertib (also known as TP-3654) for people with myelofibrosis. Myelofibrosis is a type of bone marrow disorder. The study is divided into two main parts (Phase 1 and Phase 2) and aims to check how safe nuvisertib is, if it's well-tolerated, and how it acts in the body. Researchers are giving nuvisertib either on its own or alongside another common myelofibrosis drug called ruxolitinib. It's designed for people who have intermediate or high-risk myelofibrosis, especially if their previous treatments, including JAK inhibitors, haven't worked as well as hoped, or if they can't take certain other medicines.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Sumitomo Pharma America, Inc.

Enrolment target

240

Start

16 Dec 2019

Estimated completion

30 Apr 2030

Myelofibrosis

What is this study about?

This study is designed to learn more about a new medicine called nuvisertib for people living with myelofibrosis – a condition where your bone marrow doesn't make blood cells properly. It's a two-step study: first, a 'Phase 1' part will carefully check how safe the new medicine is and if people can tolerate it, starting with small doses and slowly increasing them. Then, a 'Phase 2' part will look more closely at how well nuvisertib works, either on its own or when taken with another common myelofibrosis drug called ruxolitinib.

The main goal is to understand how nuvisertib behaves in your body and if it can help manage myelofibrosis, particularly for those whose current treatments aren't fully effective or who can't use certain other medications. The study is particularly interested in people who have already tried a type of medicine called a JAK inhibitor, but their myelofibrosis is still causing problems or their symptoms haven't improved enough. By carefully studying nuvisertib, researchers hope to find new and more effective ways to help people with this condition.

There are different groups within the study. Some people will receive nuvisertib alone because their previous JAK inhibitor treatment didn’t work or wasn't suitable. Another group will receive nuvisertib alongside ruxolitinib because they are already taking ruxolitinib but it's no longer controlling their symptoms as well as it used to. This careful approach helps the researchers understand the best way to use nuvisertib to potentially help more people with myelofibrosis.

Key takeaways

This study is testing a new medicine, nuvisertib, for myelofibrosis.
It aims to find out if nuvisertib is safe and how well it works, alone or with ruxolitinib.
It's for adults with intermediate or high-risk myelofibrosis whose current treatments aren't fully effective.
Regular clinic visits, blood tests, and scans will be part of the study.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is for adults, aged 18 or older, who have been diagnosed with myelofibrosis and whose condition is considered intermediate or high-risk.

To be considered for the study, you would generally need to have a specific type of myelofibrosis that has been confirmed by doctors. There are different groups within the study. For some groups, you might have already tried a treatment called a JAK inhibitor, but it didn't work well for you, or you weren't able to use it. For another group, you might be taking ruxolitinib for at least six months, and your dose has been steady for at least eight weeks, but it's not working as effectively as it once did. All participants must be able to move around reasonably well, have a good life expectancy, and have healthy kidney, liver, and blood clotting functions.

There are also specific blood test results needed, like having enough platelets and certain white blood cells, and your spleen must be a certain size. These checks help make sure the study medicine is safe for you and that the researchers can accurately measure any effects it might have.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of intermediate or high-risk myelofibrosis?
Have you been previously treated with a JAK inhibitor that didn't work well, or are you unable to take one?
OR are you currently taking ruxolitinib, but it's not controlling your symptoms as effectively as before?
Are your general health (kidney, liver, blood clotting) and blood counts within specific healthy ranges?
Is your spleen enlarged to a certain size?

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

What does participation involve?

If you join this study, you'll be regularly monitored by a team of doctors and nurses. This will involve regular visits to the clinic, which will include physical examinations, blood tests, and scans (like MRI or CT scans) to check the size of your spleen. You'll also be asked about your symptoms and how you are feeling using questionnaires. You will take the study medication, nuvisertib, as prescribed. Depending on which group you are in, you might take it on its own or continue taking ruxolitinib alongside it. The study will involve ongoing check-ups for a certain period to monitor your health and the effects of the treatment. The total duration of your participation in the study will depend on how your treatment progresses and the study design, but generally, clinical trials involve regular follow-up for several months or longer.

Potential risks and benefits

Participating in this study might offer a potential benefit by giving you access to a new treatment, nuvisertib, for your myelofibrosis, which your regular treatments might not be fully controlling. However, like all medicines, nuvisertib may have side effects, which could range from mild to more serious. The research team will carefully monitor you for any potential risks or side effects throughout the study. It's important to remember that joining a clinical trial is always 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 (84)

University of Alabama
Birmingham, United States· Recruiting
The University of Arizona Cancer Center
Tucson, United States· Recruiting
City of Hope
Duarte, United States· Recruiting
University of Southern California
Los Angeles, United States· Recruiting
Hoag Family Cancer Institute
Newport Beach, United States· Recruiting
Blood Cancer Center
Denver, United States· Recruiting
Yale School of Medicine
New Haven, United States· Recruiting
University of Florida Health Shands Cancer Hospital
Gainesville, United States· Completed
University of Miami
Miami, United States· Recruiting
Baptist Health - Miami Cancer Institute
Miami, United States· Recruiting
Emory University
Atlanta, United States· Recruiting
University of Chicago
Chicago, United States· Recruiting

+72 more sites — see the official record for the full list.

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.

What is a 'JAK inhibitor'?

JAK inhibitors are a type of medicine often used to treat myelofibrosis by targeting specific signals in your body that contribute to the disease.

What does 'Phase 1/2' mean for a study?

Phase 1 studies mainly check if a new drug is safe. Phase 2 studies then look at how well the drug works and continue to monitor for safety in more people.

Will I receive the new medicine?

Yes, if you meet the study requirements, you will receive nuvisertib, either on its own or alongside ruxolitinib, depending on which group you fit into.

What if my current ruxolitinib treatment isn't working as well?

One group in this study is specifically for people who are already on ruxolitinib but are seeing their symptoms worsen or not improve enough.

How to find out more

Reyna Bishop

reyna.bishop@us.sumitomo-pharma.com 617-674-6800 Official trial record

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