A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

To join this study, you must have a confirmed diagnosis of polycythemia vera (PV) and currently need regular blood removal treatments (phlebotomies) to manage your condition. You can be taking other PV medications, such as hydroxyurea, interferon, or ruxolitinib, as long as you've been on a stable dose for at least three months before the study starts. If you've stopped other PV treatments, you'd need to have done so at least three months ago, and any side effects from those treatments should have settled.

There are certain reasons why you might not be able to join. For example, if your PV has progressed to a more advanced stage called post-PV myelofibrosis, or if you have severe spleen pain. Also, if you have certain other serious health issues like active infections (including HIV, Hepatitis B or C, or current COVID-19 requiring treatment), bleeding problems, certain cancers within the last five years (unless it's a very common, easily treated type like some skin cancers), or if you've recently had major surgery, you might not be eligible.

The study is open to both men and women, aged between 18 and 90 years old.

If you decide to take part, the study will involve several stages over a significant period. First, there's a 'screening period' of up to 7 weeks to check if you're suitable. After that, the main 'treatment period' lasts for about 37 weeks (around 8-9 months), during which you'll receive the study drug. This is given as injections under your skin, with a total of 9 doses over this time. Following this, there's an 'extension period' for another 36 weeks (about 8-9 months), where you'll receive another 9 doses of the study drug.

Throughout the study, you'll be assigned either a higher or a lower dose of sapablursen, chosen randomly, but everyone will receive the active drug – there's no placebo. There will be regular visits to the clinic for assessments, blood tests, and to receive your injections. After your final dose in the extension period, there will be a 'post-treatment period' of 13 weeks (about 3 months) for follow-up and final checks. In total, your involvement could last for well over a year and a half, with regular check-ups to monitor your health and the treatment's effects.