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 may be provided.
Several research studies are evaluating an investigational medication called navitoclax for patients with myelofibrosis.
You may qualify for one of the Myelofibrosis research studies if you:
Additional study entrance criteria will apply.
An investigational medication is a medicine that has not yet been approved for use outside of this study, and it is not yet known if it is safe and effective. Navitoclax, the investigational medication being studied, is being developed to target myelofibrosis. Navitoclax was designed to target and kill cells which are not working normally in the bone marrow in 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.
Learn about research studies for a study medication targeting Myelofibrosis.
There are currently three research studies investigating Navitoclax for those with myelofibrosis. Study availability varies by country. The Myelofibrosis research studies are evaluating an investigational medication for patients with myelofibrosis. Volunteers who qualify to take part in the study may 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.
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 drug in combination with ruxolitinib are compared to the effects of the ruxolitinib 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.
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 an investigational medication, navitoclax, for patients with 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 regulatory agencies.
Each of the Myelofibrosis research studies is outlined above, along with detail as to what participating in each study might involve (please see the informational brochures associated with each study). 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 centres are conducting studies nearest to you.
Volunteers who qualify to take part in the study may receive compensation for travel. Please discuss this with the study team when you speak to them.
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:
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.
Myeloproliferative neoplasms are a type of blood cancer. To learn about blood cancers, you must first know about blood. It consists of blood cells that move within plasma. Plasma is mostly water.2
There are 3 main types of blood cells:
Your blood cells don’t live forever. Many have a short lifespan. Blood cells 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 centre of most bones. Your bone marrow 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. They 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 disrupts production of blood cells by extensive scarring in the bone marrow. When the scarring forms, the scar tissue may replace bone marrow. With less bone marrow, the number of blood cells may drop. As a result, other organs such as the spleen and liver may begin to make blood cells to try to compensate and grow larger in size. These organs may also enlarge because they trap abnormal blood cells.2
Some people 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.
The Myelofibrosis research studies are evaluating an investigational medication for patients with myelofibrosis.