IMG-7289-CTP-201
A PHASE 2 CLINICAL STUDY IN PATIENTS WITH ESSENTIAL THROMBOCYTHEMIA
IF YOU ARE INTERESTED IN LEARNING MORE ABOUT THE STUDY, PLEASE VISIT WWW.CLINICALTRIALS.GOV: NCT04254978
About Essential Thrombocythemia
Essential thrombocythemia (ET) is one of a family of rare bone marrow diseases called myeloproliferative neoplasms (MPNs).
Bone marrow, the spongy tissue within the hollow portion of the bones, contains specialized cells that make platelets (cells that help your blood clot). ET is a condition in which the bone marrow makes too many platelets that can lead to abnormal blood clots or bleeding. Patients with ET may experience symptoms including fatigue, weight loss and a decrease in appetite that can make their quality of life worse. Rarely, patients with ET develop bone marrow failure or leukemia.
What is IMG-7289?
Bomedemstat (IMG-7289) is a new investigational medication, meaning it has not been approved by the U.S. Food and Drug Administration (FDA), taken daily by mouth. The FDA has granted Fast Track designation to bomedemstat for the treatment of essential thrombocythemia. The goal of this treatment is to reduce the activity of the bone marrow cells that make platelets. Bomedemstat has been studied in patients with other diseases of the bone marrow. This is the first Imago study using bomedemstat for the treatment of ET.
What is the IMG-7289-CTP-201 study?
The IMG-7289-CTP-201 study is a Phase 2 study in patients with a diagnosis of ET. To enter this study, patients must have a platelet count above the normal range in need of treatment. Patients must also have been treated with another platelet-lowering drug that no longer benefits them.
Participants will need to visit a study site to see if they meet the criteria for the study.
The initial treatment period with bomedemstat is 24 weeks (about 6 months), during which, participants will visit the clinic about 10 times. There will be regular physical examinations and laboratory tests such as blood counts. In addition, there will be bone marrow samples taken before the start of treatment and at Week 24.
Participants receiving clinical benefit from bomedemstat can continue treatment beyond 24 weeks.
Who Can Take Part in IMG-7289-CTP-201 study?
You may be eligible for the study if you:
- Are over the age of 18
- Have been diagnosed with Essential Thrombocythemia
- Require treatment in order to lower platelet count (based on patient age over 60 or history of thrombosis)
- Have failed at least one standard therapy
- Will discontinue any current ET therapy at least 1 week (4 weeks for interferon) prior to taking bomedemstat.
Trial Locations:
This study is being conducted at sites in the United States, United Kingdom, Australia, New Zealand, Germany and Italy. For additional information about the study, visit ClinicalTrials.gov and enter the study identifier (NCT number: NCT04254978)
Essential Thrombocythemia Clinical Trial Site FAQ
Essential Thrombocythemia Clinical Trial Site Frequently Asked Questions (FAQ)
What Are Clinical Trials?
- Clinical trials are studies of investigational drugs, known as study drugs, in healthy volunteers or patients. ‘Investigational’ means the drug has not been approved by the U.S. Food and Drug Administration (FDA). Clinical trials are performed to better understand how drugs behave in the body and how the body reacts to them.
Pharmaceutical companies must test their study drugs in a series of studies known as clinical trials before requesting permission from the FDA for doctors to be able to prescribe them. Depending on the type of drug being studied, healthy volunteers or patients willingly participate in these trials to establish their safety and efficacy. - Each trial follows a detailed plan (or “Protocol”) which explains all activities that occur during the trial. Additionally, clinical investigators including nurses and doctors must explain the trial to the patients and answer completely all questions patients ask. In the United States, clinical trial protocols must be reviewed and approved by the FDA and also by an independent group called an Institutional Review Board (IRB) established to ensure the protection of the rights of research participants. The IRB does not work for the pharmaceutical company or the doctor performing the trial. Members of the IRB are scientists, doctors, lawyers and members of the public.
- Healthy volunteers and patients participate in trials because it is possible they could benefit from the study drug. Some take the view that participation may in the future help others with the condition. Participants can also access medical care options and diagnostic tools that might not otherwise be available to them.
- With any medical research study there are risks associated with participation. There is always the possibility that the study drug may not have the desired effect on the disease being treated. Side effects are also possible. Doctors assess the potential benefits of the study drug and the risks of side effects based on previous studies and on animal studies conducted before a drug is given to humans. Doctors discuss the specific risks involved with participating in a clinical trial before allowing someone to join.
- There are four phases of clinical trials. The trials at each phase have a different purpose and help answer different questions.
- Phase 1: Test a study drug in a small group of healthy volunteers (people without the condition) for the first time to evaluate the treatment’s safety, how much can be given safely (what’s the right dose?), and to identify possible side effects.
- Phase 2: Test a study drug in a larger group of people – participant are always patients with the condition – to see if the treatment is effective and to further evaluate the safety of the treatment. In some studies, patients may be given either the study drug or placebo without knowing which one they are taking.
- Phase 3: Test a study drug in a large group of patients to confirm if the treatment is effective, to monitor side effects, to compare the treatment with other treatments, and to collect additional information which should help allow safe use of the treatment. In most Phase 3 clinical trials, patients are usually given either active medication or an inactive placebo without knowing which one they are taking.
- Phase 4: Studies conducted once a drug is approved and, on the market, to establish additional information about the broader risks, benefits, and best use of the treatment.
- The number of people included in each stage of a trial increases from one stage to the next, with the number in any given trial depending on the disease the trial is studying.
- Fast Track Designation is a label given by the U.S. Food and Drug Administration (FDA) to study drugs having the potential to treat a serious condition where there is an unmet medical need. Bomedemstat has received Fast Track Designation from the FDA for the treatment of ET. This Fast Track Designation allows the biopharmaceutical company to work closely with FDA to potentially speed up the availability of new therapies to patients.
- ET is part of the family of rare bone marrow diseases called Myeloproliferative Neoplasms (MPNs).
- ET is a condition in which the bone marrow makes too many platelets that can lead to abnormal blood clots or bleeding.
- In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infarctions (tissue death due to a lack of blood supply to the affected area), each having a significant impact on these patients.
- Patients with ET can develop bone marrow failure or leukemia, though this is rare.
- Fewer than 1 in 100,000 patients are diagnosed with ET in any year (National Organization for Rare Diseases).
- ET is a quiet bone marrow cancer than can linger for years. Patients with ET can experience symptoms including fatigue, weight loss and a decrease in appetite that can make their quality of life worse. Who is Conducting this Clinical Study?
- Imago BioSciences is a company focused on developing medicines for the treatment of bone marrow disorders including essential thrombocythemia (ET), myelofibrosis (MF) and polycythemia vera (PV). Imago BioSciences is conducting this clinical trial with the study drug bomedemstat (also known as IMG-7289). The company was founded in 2012 and is based in California. For more information, visit www.imagobio.com.