Patients, Caregivers and Advocates
Frequently Asked Questions

What is Polycythaemia Vera (PV)?
PV is a rare blood cancer characterised by an excess production of red blood cells2. PV affects approximately 3.5 million people worldwide3.
How is PV currently treated?
The current standard of care includes a procedure called a phlebotomy that reduces the percentage of red blood cells in the blood, and/or medications to reduce red blood cell counts. There is currently no cure for PV and people living with the disease are at an increased risk of cardiovascular events and potential progression to myelofibrosis or leukemia2,4,5.
What is SLN124 and how does it work?
SLN124 works by temporarily silencing the production of the TMPRSS6 protein found in liver cells, which in turn increases the body’s production of another protein called hepcidin. By increasing hepcidin levels, SLN124 aims to redirect iron delivery away from the bone marrow, lowering red blood cell production and potentially reducing the high red blood cell count in people living with PV. This may reduce the need for phlebotomies and lower the risk of blood clots and strokes. In addition, PV related symptoms such as fatigue, brain fog, and itchy skin may be reduced.
What is an siRNA?
siRNA is a short interfering RNA. siRNA therapeutics work by leveraging the body’s natural process of RNA interference and aim to treat disease by “silencing” the genes that cause or contribute to them. Unlike gene therapy or editing, it does not produce permanent changes.
What is a clinical study?
A clinical trial is a research study where experts study potential treatments. The treatments might be new drugs or devices. Clinical trials, also known as clinical studies, must meet specific standards and regulations. Should you decide to join a clinical study, know that your well-being is the clinical study team’s top priority. And you can leave a study at any time.
Why are clinical studies important?
Clinical studies are essential for understanding whether new treatments are safe and effective. They help advance medical research and can lead to better care in the future. Participation is voluntary and personal—many people join to help others with the same condition or to find a better treatment or to access a potential new therapy.
What are the different phases of clinical studies?
There are typically four main phases of clinical studies (phase 1 to phase 4). Each phase has a specific purpose and study design. A phase 1 study focuses on evaluating the safety, dose range and side effects of a medication. A phase 2 study assesses the effectiveness of a medication and further evaluates the safety profile. A phase 3 study is used to confirm the effectiveness of the medication, monitor side effects, and compare the medication to standard treatments. Finally, a phase 4 study is typically conducted after a medication has received regulatory approval and is used to monitor the long-term effectiveness and safety of the new medication.
Where will the SANRECO clinical study take place?
The SANRECO study currently has 19 locations worldwide. Please click here to view all study locations.
What are the costs for participating in the SANRECO study?
There is no cost associated with participating in this study. Travel arrangements (such as transportation) will be provided. You will also be reimbursed for expenses that are related to your study visits, such as refreshments and parking. Accommodation will be provided where needed.
How do I know if I or a loved one is eligible to participate in the SANRECO study?
In order to participate in the SANRECO study, you must be at least 18 years of age, have been clinically diagnosed with polycythaemia vera and had 3 or more phlebotomies in the last 7 months, or 5 or more in the last 12 months. Additional criteria may also be required. If you are interested in learning more, please complete this form.
What if I experience an adverse event while in the SANRECO study?
If you experience an adverse event during a clinical study, report it immediately to the study staff and seek medical attention if needed. You have the right to withdraw from the study at any time and may be entitled to care or compensation, as outlined in your informed consent form.
How do I find out more information about participating in the SANRECO study?
Please click here to contact us if you would like more information about the SANRECO study.
2. NORD Rare Disease Database, Polycythaemia Vera. Treatment goal is to control HCT <45% to reduce CV and major thrombotic events https://rarediseases.org/rare-diseases/polycythemia-vera/
3. Using 44/100,000 global population: 7,800m, Kattamis, A. et al. European Journal of Haematology. 2020; 105(6): 692-703.
4. Spivak, Jerry L. Polycythaemia vera. Current treatment options in oncology. 2018: (19)1-14.
5. Marchioli, Roberto, et al. Cardiovascular events and intensity of treatment in polycythaemia vera. New England Journal of Medicine. 2013; 368.1: 22-33.