Disease information
Polycythemia Vera is a blood disorder in which the bone marrow in the body produces too many red blood cells. Patients with PV are at increased risk of developing blood clots that may cause heart attacks and strokes. The symptoms of PV can vary but include headache, itching, dizziness, and lack of energy.
The diagnosis of PV consists of a blood draw and can indicate if the patient has elevated levels of red blood cells and hemoglobin. Furthermore, a bone marrow biopsy can be collected.
Therapeutic background
As there is currently no cure for PV, treatment focuses on controlling the volume of red blood cells (called hematocrit levels) to reduce the risk of blood clots. The standard therapeutic goal is to maintain hematocrit levels below 45%. One type of treatment is called phlebotomy, which is a procedure in which blood is removed to reduce the number of red blood cells in the body. Com- mon drug treatments used alone or in combination with phlebotomy are:
- Hydroxyurea
- Janus Kinase (JAK) inhibitor
- Interfero
Unfortunately, for some patients, the current PV treatment options are not fully effective or are not tolerated or may cause serious side effects. Therefore, new therapies to maintain the hematocrit levels, are also being investigated.
Who can participate?
Candidates who fulfil the following criteria may be eligible for the study:
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Male or females aged 18 years or older.
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Diagnosed with Polycythemia vera
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Requires phlebotomies to control PV hematocrit levels (e.g., at least 3 phlebotomies in the last 6 months or at least 5 in the last year)
Locations
Reimbursement
There will be no costs to you if you choose to participate in this research study. The investigational medication and any tests required by the study are provided by the study team. You may receive reimbursement for travel expenses incurred from taking part in the research study.
Study description
The aim of the study is to evaluate a new investigational treatment for safety and its ability to control the number of red blood cells, to reduce, delay or eliminate phlebotomy and to improve patients’ quality of life. The study is divided into different periods.
Screening period
The screening period will last approximately 28 days. It is conducted to confirm your eligibility for the research study based on clinical or laboratory assessments performed at a local study site near you. At the screening visit, you will be asked to sign a form that indicates you are willing to participate in the study. Study staff will also ask you questions about your health. More detailed information will be provided when you visit the site.
Treatment period
If you are eligible to participate in the study after the screening period, you will enter a study treatment period. Participation in the study is divided into three parts and can last up to 166 weeks (slightly over 3 years).
As a study participant, you can expect to:
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Complete a screening visit with assessments to confirm you meet the study requirements.
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Continue your current PV therapy.
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Receive injections every week under the skin for up to 3 years. You will receive initial doses by the study team during a visit. You will be trained to do the injections and when you are comfortable, you will do the weekly injections at home.
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Depending on how long you have been in the study, you will visit the study clinic every 2, 4, 6, or 12 weeks for study assessments.
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Attend 1 follow-up visit at the study clinic 4 weeks after your final dose of study drug.
The study layout below explains the 3 parts of the study in more detail:
Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
