Active not recruitingPHASE1INTERVENTIONAL

Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms

This study is investigating a new medication called INCB057643. We want to find out how safe it is, what side effects it might cause, and how well it works for people with myelofibrosis and other related blood conditions. The study will test INCB057643 on its own or in combination with another common treatment, ruxolitinib. This is an early-stage study, meaning it's one of the first times this new drug is being tested in people. The main goals are to understand its safety and find the most suitable dose, with an initial look at whether it helps improve the condition.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

Incyte Corporation

Enrolment target

140

Start

23 Feb 2021

Estimated completion

30 Apr 2027

Myelofibrosis Myelodysplastic Syndrome Myelodysplastic/Myeloproliferative Neoplasm Overlap Syndrome Myeloproliferative Neoplasm Relapsed or Refractory Primary Myelofibrosis Secondary Myelofibrosis (Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis)ET (Essential Thrombocythemia)

What is this study about?

This research study is focused on a new drug, INCB057643, for people living with specific types of blood disorders. These conditions, such as myelofibrosis, myelodysplastic syndrome (MDS), and essential thrombocythemia (ET), affect how your blood cells are made in the bone marrow. They can cause symptoms like tiredness, feeling unwell, and an enlarged spleen.

The main aim of this study is to carefully check if INCB057643 is safe and well-tolerated. This means understanding any side effects people might experience and finding the right amount of the drug to give. We'll also be looking to see if the drug shows any early signs of helping to manage these conditions, either when given by itself or when combined with ruxolitinib, which is a medicine already used for some of these conditions.

Finding new and effective treatments is really important for people with these conditions. This study is an important step in figuring out if INCB057643 could be a helpful option in the future. By taking part, volunteers contribute valuable information that could lead to better care for others.

Key takeaways

This study is testing a new drug, INCB057643, for specific blood cancers.
It aims to find out how safe the drug is and the best dose to use.
The drug might be given alone or with an existing treatment, ruxolitinib.
It's for adults (18+) with conditions like myelofibrosis or myelodysplastic syndrome.
Participants will have regular check-ups and monitoring.
You can stop participating at any time.

Who may be eligible?

This study is looking for adults aged 18 or older who have specific blood conditions. These include myelofibrosis (primary or after polycythemia vera/essential thrombocythemia), myelodysplastic syndrome, myelodysplastic/myeloproliferative overlap syndrome, or essential thrombocythemia.

For some parts of the study, you might be eligible if your current treatment for these conditions isn't working well enough, or if you've already tried at least one other treatment. For example, if you have myelofibrosis, you'd typically need to have had a specific type of treatment called a JAK inhibitor (like ruxolitinib) before. If you have essential thrombocythemia, your condition would need to have not responded well to a medicine called hydroxyurea.

Another group of people who might be suitable are those with myelofibrosis who are either already taking ruxolitinib but not getting enough benefit, or those who have never taken a JAK inhibitor before. The medical team will check other specific details about your condition to see if you meet all the requirements for this study.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of myelofibrosis, myelodysplastic syndrome (MDS), or essential thrombocythemia (ET)?
Have you tried at least one other treatment for your condition, or is your current treatment not working well enough?
If you have myelofibrosis, have you previously taken a JAK inhibitor like ruxolitinib, or are you currently taking it but not seeing enough benefit, or have never taken one before?
If you have ET, has your condition not responded well to hydroxyurea?

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 the new study drug, INCB057643. Depending on which part of the study you join, you might receive INCB057643 on its own or together with another medicine called ruxolitinib. You will have regular visits to the study clinic where the doctors and nurses will closely monitor your health, take blood and other samples, and ask you about how you are feeling and any side effects you might be experiencing. These visits are important to check your progress and ensure your safety. The total duration of your participation in the study will depend on the specific part of the study you are in and how you respond to the treatment, but it will involve multiple appointments over a period of time.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that the new drug, INCB057643, might help to manage your condition or improve your symptoms. However, as this is a new medicine, there's no guarantee it will work for you. There are also potential risks, as INCB057643 might cause side effects, some of which could be serious or unexpected. The study team will explain all known and potential side effects to you. It's important to remember that you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (48)

University of Alabama At Birmingham
Birmingham, United States
University of Colorado Cancer Center
Aurora, United States
University of Miami Sylvester Comprehensive Cancer Center
Miami, United States
Emory University-Winship Cancer Institute
Atlanta, United States
University of Iowa Hospital and Clinics
Iowa City, United States
Washington University School of Medicine
St Louis, United States
Rutgers Cancer Institute of Nj
New Brunswick, United States
Nyu Langone Health - Long Island Hospital
Mineola, United States
Nyu Langone Laura and Isaac Perlmutter Cancer Center
New York, United States
Weill Medical College of Cornell University
New York, United States
University of North Carolina At Chapel Hill
Chapel Hill, United States
University of Cincinnati Cancer Institute
Cincinnati, United States

+36 more sites — see the official record for the full list.

Common questions

What is myelofibrosis?

Myelofibrosis is a blood disorder where scar tissue builds up in your bone marrow, affecting how your body makes blood cells.

What is a Phase 1 study?

A Phase 1 study is an early stage of clinical research. Its main goal is to check how safe a new drug is and find the right dose, rather than primarily seeing how well it works.

What is INCB057643?

INCB057643 is a new, experimental drug being tested in this study to see if it can help people with certain blood conditions like myelofibrosis.

Will I get the new drug or a placebo?

In this specific study, participants will receive the active drug INCB057643, either on its own or with ruxolitinib. This study does not use a placebo.

What if my condition gets worse during the study?

Your health will be closely monitored throughout the study. If your condition worsens or if you experience severe side effects, the study doctors will stop the study drug and discuss the best next steps for your care.