Have you considered a myelofibrosis research study as part of your treatment plan for your diagnosis of myelofibrosis?

The primary objective of the study is to evaluate the effect of navitoclax in combination with ruxolitinib in people with myelofibrosis.

If you are eligible to participate, you may be helping to find possible treatment options for myelofibrosis, potentially helping you and others in the future.

This research study is evaluating a new investigational medication, called navitoclax, for myelofibrosis.

You may qualify for the myelofibrosis research study if you:

  • Are over 18 years old
  • Have a diagnosis of myelofibrosis
  • Are interested in participating in a clinical research study

Additional study criteria will apply.

About the Investigational Medication

An investigational medication is a product that is being studied in the clinic whose safety and efficacy are under evaluation.

The investigational medication has not yet been approved by global regulatory health authorities. Navitoclax, the investigational medication being studied, is being developed to target myelofibrosis. Navitoclax was designed to target and kill the cancer cells which are not working normally in the bone marrow in people with myelofibrosis.

Navitoclax is being evaluated in this study to evaluate its effect on splenomegaly (enlarged spleen), bone marrow fibrosis, and anemia responses as well as quality of life.

Navitoclax has not been approved by regulatory health authorities for people living with myelofibrosis. Safety and efficacy are under evaluation.

All study-related care will be provided by a team of medical professionals.

Volunteers who are eligible to take part in the Myelofibrosis research study may receive reimbursement for travel.


About the Myelofibrosis Research Study

Learn about the research study for a new investigational medication targeting myelofibrosis.

There were previously ongoing research studies investigating navitoclax for those with myelofibrosis. This myelofibrosis research study is evaluating a new investigational medication for people with myelofibrosis.

Navitoclax has not been approved by regulatory health authorities. Safety and efficacy are under evaluation. During this study, researchers need to confirm the effectiveness of the investigational medication and monitor its side effects in a large group of study participants living with myelofibrosis.

All study-related care is provided by a team of experienced specialist doctors and nurses. Volunteers who qualify to take part in the study may receive compensation for travel.

Transform2 H-20-178

In the TRANSFORM-2 study the investigational medication (navitoclax) in combination with ruxolitinib is compared to a standard therapy to evaluate its effect on splenomegaly (enlarged spleen), bone marrow fibrosis (development of scar tissue within the bone marrow which can impact the healthy tissue), anemia (condition of having a lower than normal red blood cell count) responses and quality of life (your overall wellbeing) in patients that have received previous treatment for their myelofibrosis.

This study is being conducted globally at approximately 210 research centers and there will be approximately 330 patients with myelofibrosis who will participate in this study.

You may be eligible for the TRANSFORM-2 study if you:

  • Are 18 years of age or older
  • Have been diagnosed with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis
  • Currently have Intermediate-2 or High-Risk myelofibrosis
  • Are currently taking or have received prior treatment with ruxolitinib
  • Have splenomegaly (enlarged spleen)
  • Are experiencing symptoms related to myelofibrosis despite current or prior ruxolitinib treatment

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of treatment will be checked by medical assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.

The ClinicalTrials.gov identifier (NCT number) is NCT0446898.

Who can take part
in the Myelofibrosis
research study?

If you meet these criteria and are interested in more information, please contact your doctor to discuss the myelofibrosis research study to see if you are eligible to participate. You should ask your doctor if you have any questions about the investigational medication, including potential side effects and standard treatments that are available for myelofibrosis. Your doctor may also speak to the study physician to ask questions about this research study to see if it may be right for you.

The myelofibrosis research study is being conducted at research centers across the globe. Please use the map below to locate a research center in your area.

Learn more about how to enroll by contacting our concierge enrollment service powered by Massive Bio.
Call 1-855-M2-STUDY | 1-855-627-8839 or click below for enrollment information.


Frequently Asked Questions

What is a clinical research study?

A clinical research study (also called a clinical trial) is a scientific study that evaluates how safe and effective an investigational medication is working for a certain condition. All medications must be tested in clinical research studies before they can be approved and prescribed to patients.

The myelofibrosis research study is evaluating a new investigational medication, navitoclax, for myelofibrosis.

During this study, researchers will be evaluating how safe and well the investigational medication works and monitor its side effects in the group of study participants. Navitoclax has not been approved by the FDA, European Medicines Agency (EMA) or other regulatory health authorities. It’s safety and efficacy are being evaluated.

The myelofibrosis research study is outlined above, along with detail as to what participating in this study might involve. The length of time you may spend in the study may be for as long as you are receiving benefit. If you decide to participate and you are eligible for a study, the study team will closely monitor your health and protect your privacy. Your participation in the study is voluntary – you can stop participating at any time for any reason.

Volunteers who qualify to take part in the study may receive reimbursement for travel. Please discuss this with the study team when you speak to them. If you need assistance with travel, please discuss with the study site staff.

There is no cost to participate in the myelofibrosis research study. If you decide to take part, you will receive study-related care throughout the study from a team of experienced doctors and nurses.

The research team will be able to explain more about what the myelofibrosis research study will involve, and it is up to you to decide if you want to take part. Participation in a study is voluntary. Whether or not you decide to participate in a study will not affect your current or future relationships with your doctors. If you decide to participate, you are free to withdraw at any time without affecting those relationships.

There may be a research center within a close travel distance from your home. If we are not conducting the study in your area currently, please check back regularly to see if a study in your area becomes available.

About Myelofibrosis and Blood Cancers

Myelofibrosis is part of a group of diseases called myeloproliferative neoplasms - these are a type of blood cancer. To understand blood cancers, let’s first take a look at blood itself. Blood consists of blood cells that move within plasma, which is mostly water.2

There are 3 main types of blood cells:

  • Red blood cells (also called erythrocytes)
  • Platelets (thrombocytes)
  • White blood cells (leukocytes) which include granulocytes, monocytes, and lymphocytes

Your blood cells don’t live forever. Many have a short lifespan and are being replaced in your body all the time.

Most blood cells are formed in bone marrow. Bone marrow is the sponge-like tissue in the center of most bones which contains blood-forming cells. These cells are called blood stem cells or hematopoietic stem cells. They are the cells from which all blood cells are formed.2

Blood cancers are a group of cancers. These cancers form from blood cells in bone marrow. Blood cancers include leukemia (abnormal stem cells and/or abnormal white blood cells), myelodysplastic syndromes (abnormal stem cells leading to fewer and abnormal blood cells), and myeloproliferative neoplasms (abnormal stem cells leading to more blood cells than normal). One of the myeloproliferative neoplasms is myelofibrosis (MF).

Myelofibrosis is a serious, rare bone marrow disorder that causes extensive scarring in the bone marrow disrupting the production of blood cells. When the scarring forms, the scar tissue may replace bone marrow. With less bone marrow, the number of blood cells the bone marrow can produce may drop. As a result, other organs such as the spleen and liver may begin to make blood cells to try to compensate. The added stress on the liver and spleen may cause them to grow larger in size. These organs may also enlarge because they trap abnormal blood cells.2

Some people with myelofibrosis may not show any symptoms at all, and the people that do show symptoms may experience them differently. These symptoms include:1

  • Feeling tired or weak
  • Bruising easily
  • Bleeding easily
  • Pain in the ribs
  • Fever
  • Bone pain

Current treatments focus on relieving the symptoms of myelofibrosis. Treatment also aims to improve the number of blood cells in the blood and reduce the chance of the disease getting worse. Myelofibrosis research continues to focus on studying the progression of the disorder as well as potential treatment options.

The myelofibrosis research study is evaluating a new investigational medication for myelofibrosis.

What happens if I want to learn more?

You will visit a study location in your area where the myelofibrosis research study is currently being conducted.

If you think you might be interested in participating in the myelofibrosis research study, or wish to learn more about the study, contact a research center near your location so we can see if you may qualify. Keep in mind that participation is entirely voluntary. If you are eligible and do decide to take part in a study, you may change your mind about participating at any time. Additional study entrance criteria will be evaluated by the study staff at the research center.