A Study of Rusfertide in Japanese Adults With Polycythemia Vera (NCT07648030) | Clinical Trial Compass
Not Yet RecruitingPhase 2
A Study of Rusfertide in Japanese Adults With Polycythemia Vera
9 participantsStarted 2026-06-23
Plain-language summary
Polycythemia vera (PV) is a rare blood cancer in which the body makes too many red blood cells. This can make the blood thicker and may increase the risk of serious health problems such as blood clots. Many people with PV need regular phlebotomy, which is a procedure to remove blood, to help keep their hematocrit level under control. Hematocrit is the proportion of red blood cells in the blood.
The main aim of this study is to evaluate whether rusfertide helps Japanese participants with PV keep their hematocrit under control and avoid the need for phlebotomy. All participants in this study will receive rusfertide. This study is open-label, which means both the participants and the study team will know what treatment is being given.
Participants will be followed for up to about 244 weeks, including a screening period, a 52-week initial treatment period, a long-term extension period, and a 4-week safety follow-up period.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Japanese male and female participants aged 18 years or older at the time of signing informed consent.
. Participant understands the trial procedures, is willing and able to adhere to trial requirements and agrees to participate in the trial by giving written informed consent.
. Meet revised 2016 WHO criteria for the diagnosis of PV.
. Phlebotomy requiring defined as ALL of the following:
. At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before trial intervention or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before trial intervention, and
. Last phlebotomy due to inadequate hematocrit control within 3 months before trial intervention, and
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of Participants Achieving a Response Starting at Week 20 through Week 32
. No phlebotomy within 6 days prior to trial intervention (do not include day of phlebotomy and day of trial intervention in the 6-day count).
. Hematology test values at screening:
Exclusion criteria
. Clinically meaningful laboratory abnormalities at Screening including, but not limited to:
. eGFR \<15 mL/min/1.73 m\^2 as determined by Japanese Society of Nephrology. Calculated by the correction formula for Japanese\*
. ALT or AST ≥2.5×ULN
. Total bilirubin \>1.5×ULN. Note: Screening laboratory tests with abnormal results (if considered by the investigator to be transient and inconsistent with the participant's clinical condition) may be repeated within the screening window to confirm abnormal results. If results return to protocol acceptable limits within the screening period, the participant may enter the trial. Use local labs for all eligibility lab tests.
. Participants who require phlebotomy at hematocrit levels lower than 45%.
. Pregnant females will be ineligible to participate in this trial if, despite a negative pregnancy test, the investigator determines that based on interview, clinical assessment, or other relevant information that the individual may be in the very early stages of pregnancy.
. Is capable of breastfeeding but does not agree to forego breastfeeding from the first dose of trial intervention through 30 days after the last dose of trial intervention.
. Clinically significant thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to trial intervention.