A Study of Rusfertide in Adults With Polycythemia Vera in China (NCT07765602) | Clinical Trial Compass
Not Yet RecruitingPhase 2
A Study of Rusfertide in Adults With Polycythemia Vera in China
24 participantsStarted 2027-03-01
Plain-language summary
Polycythemia vera (PV) is a long-term condition in which the bone marrow makes too many red blood cells (RBCs). Bone marrow is the soft tissue inside the large bones where blood cells are made. When there are too many RBCs, called erythrocytosis, the blood can become thicker and move less easily through blood vessels and organs. This can raise the risk of blood clots (thrombosis) in veins or arteries. Sometimes, these clots can be life-threatening and may cause a stroke or heart attack. Treatment aims to keep the percentage of RBCs in the blood (hematocrit) in a safe range. For adults with PV, this means keeping the hematocrit below 45%. This helps lower the blood thickness and the risk of thrombosis. PV can also be linked to low iron levels (iron deficiency), because the body uses iron to make more RBCs. Many people with PV are treated with blood removal (phlebotomy) to lower RBC levels in the blood. This can worsen the iron deficiency, because each blood removal also takes iron out of the body.
Rusfertide is a medicine that lowers the amount of iron available in the blood. It works like hepcidin, a natural hormone that helps control iron levels in the body. By limiting iron available for RBC production, rusfertide may help keep hematocrit below 45% and may improve symptoms. It may also reduce, or even remove, the need for phlebotomy in people.
The main aim of this study is to check how well rusfertide works to lower the number of phlebotomies needed in Chinese adults with PV. Other aims are to understand how well rusfertide works to keep hematocrit levels under control and how safe it is. The study also wants to learn how rusfertide moves through the body (pharmacokinetics) and if it causes the body's defense system to react to it (immunogenicity).
During the study, participants will receive rusfertide for up to 1 year (52 weeks) and will have to visit their study clinic several times. Blood samples will be taken several times during the study.
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
. Chinese male and female participants aged 18 years or older at the time of signing of informed consent.
. Participant understands the trial procedures, is willing and able to adhere to trial requirements, and agrees to participate in the trial by providing written informed consent.
. Meets the revised 2016 World Health Organization criteria for the diagnosis of polycythemia vera (PV).
. Participant with inadequate hematocrit control who meets all of the following criteria:
. Greater than or equal to (≥) 3 times documented hematocrit ≥45 percent (%) within 28 weeks prior to trial intervention or ≥5 times documented hematocrit ≥ 45% hematocrit within 1 year prior to trial intervention.
. Documented hematocrit ≥45% within 3 months prior to trial intervention.
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
. Phlebotomy or erythrocytapheresis, if performed, must not have occurred within 6 days of trial intervention administration (the day of phlebotomy or erythrocytapheresis and the day of trial intervention administration should not be included in the 6-day count).
. Complete blood count immediately prior to trial intervention administration must meet the following criteria:
Exclusion criteria
. Clinically significant laboratory abnormalities at screening, including but not limited to:
. Estimated glomerular filtration rate (eGFR): \<15 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) according to the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation 2021. Estimated Glomerular Filtration Rate (eGFR) =142 × (serum creatinine \[Scr\] / A)\^B × 0.9938\^age × (1.012 if female), where A and B are the following: Female: Scr less than equal to (≤) 0.7, A equals to (=) 0.7, B=-0.241; Scr greater than (\>) 0.7, A=0.7, B=-1.2. Male: Scr ≤0.9, A=0.9, B=-0.302; Scr \>0.9, A=0.9, B=-1.2. Creatinine unit conversion: milligrams per deciliter (mg/dL) =micromoles per liter (μmol/L)/88.4.
. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5×upper limit of normal (ULN).
. Total bilirubin \>1.5×ULN.
. Those who require phlebotomy at hematocrit levels \<45%.
. Clinically significant thrombosis (for example, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to trial intervention.
. Active or chronic bleeding within 2 months prior to trial intervention.
. Those who meet the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment.