The primary objective of the studies 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. All study-related visits, tests, and investigational medication will be provided at no cost. In addition, compensation for travel will be provided.
Several research studies are evaluating an investigational new treatment option, called navitoclax, for myelofibrosis.
You may qualify for one of the Myelofibrosis research studies if you:
Additional study criteria will apply.
About the Investigational Medication
An investigational medication is a product that has not yet been approved for use by regulatory health authorities. Safety and efficacy are under evaluation. 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 several studies 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.
Learn about research studies for new investigational medication targeting Myelofibrosis.
There are currently two research studies investigating Navitoclax for those with myelofibrosis. The Myelofibrosis research studies are evaluating a new investigational medication for people with myelofibrosis. Volunteers who qualify to take part in the study will receive compensation for travel.
Navitoclax has not been approved by regulatory agencies. 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.
In the TRANSFORM-1 study, navitoclax in combination with ruxolitinib will be compared to ruxolitinib alone 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), and anemia (condition of having a lower-than-normal red blood cell count) responses and quality of life (your overall wellbeing).
In the TRANSFORM-1 study, all patients will receive ruxolitinib. Some patients will receive the investigational drug, navitoclax, in combination with ruxolitinib, and some patients will receive a placebo tablet or pill (an inactive pill that has no treatment value) in combination with ruxolitinib. The effects of the investigational medication in combination with ruxolitinib are compared to the effects of the ruxolitinib in this study.
This study is being conducted globally at approximately 190 research centers and there will be approximately 230 subjects with myelofibrosis who will participate in this study.
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.
The Myelofibrosis research studies are being conducted at research centers across the globe. Please use the map below to locate a research center in your area.
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 studies are evaluating a new investigational medication option, 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.
Each of the Myelofibrosis research studies is outlined above, along with detail as to what participating in each 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.
You do not need to know which study is right for you. We will help identify which centers are conducting studies nearest to you. To learn if there is a study center near you, you can complete the online questionnaire here.
Volunteers who qualify to take part in the study will receive compensation 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 studies. If you qualify, all study-related visits, tests, and investigational medication will be provided at no cost. If you decide to take part:
Neither you nor your insurance company will have to pay for the study medication or procedures that are done only for the study. You or your insurance company will be responsible for the costs of your regular medical care. Regular medical care costs are the costs for medications, treatments, procedures, and testing that you would have had if you were not in the study.
The research team will be able to explain more about what each of the Myelofibrosis research studies 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.
We match you to a research center within a close travel distance from your home. If we are not conducting the study in your area currently, with your permission, we will keep you in our database and reach out once a study in your area becomes available. If, at any time, you decide you no longer want your information stored, you can opt out and we will delete your details.
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:
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
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 studies are evaluating a new investigational medication for myelofibrosis.
What happens if I complete the information on the questionnaire? You will be matched to a study location in your area where one of the Myelofibrosis research studies is currently being conducted.
If you think you might be interested in participating in a Myelofibrosis research study, or wish to learn more about the studies, please enter your information below 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.