A Study of Rusfertide in Adults With Polycythemia Vera in China
ConditionPolycythemia Vera
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorTakeda
About this trial
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.
Eligibility criteria
Qualifiers
1. 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).
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.
Disqualifiers
Estimated glomerular filtration rate (eGFR): <15 milliliters per minute per 1.73 square meters (mL/min/1.73 m²) 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%.
Trial design
Treatments tested in this trial
- Rusfertide
Treatment groups
24 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov