A Study of Rusfertide in Adults With Polycythemia Vera (PV)

To join this study, you need to be at least 18 years old and have a confirmed diagnosis of polycythemia vera (PV) according to specific medical guidelines. A key requirement is that your PV has recently needed frequent blood removal (phlebotomy) to control your blood thickness, specifically at least three times in the last six months or five times in the last year.

Your blood test results, including your red blood cell count, white blood cell count, and platelet count, need to be within certain ranges just before starting the study. If you are already taking other medicines for your PV, these treatments need to have been stable for a while. If you manage your PV with phlebotomy alone, you should not have taken other PV-specific medicines within 2 to 6 months before the study starts.

There are also some reasons why you might not be able to join. For example, if you have other serious health problems, have had recent blood clots, or have active bleeding. You also can't join if you’ve had certain types of cancer in the last five years (unless fully treated and localised, like some skin or prostate cancers), or if you’ve taken certain specific PV medicines within the last seven months.

If you decide to take part, the study will last for a long time. For the first 32 weeks, you will receive either the study medicine (rusfertide) or a dummy medicine (placebo), in addition to your usual PV treatment. This phase is 'double-blind', meaning neither you nor your study doctor will know which one you are receiving. During this time, you will have regular visits to the clinic for check-ups, blood tests, and to get your study medicine.

After these 32 weeks, if you successfully complete this part of the study, you will then receive rusfertide for a much longer period – about 124 weeks (over two years). This ensures everyone gets a chance to take the active medicine. Throughout these phases, you will continue to have regular clinic visits to monitor your health, blood counts, and any side effects. After your last dose of rusfertide, the study team will contact you by phone around 6 months and 12 months later to check on your well-being. The total duration of active participation where you are taking medication and having clinic visits will be approximately 156 weeks (32 + 124).