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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Rusfertide injections administered SC.
Percentage of Participants Achieving a Response Starting at Week 20 Through Week 32
Response is defined as the absence of phlebotomy eligibility. Phlebotomy eligibility is defined as: A confirmed hematocrit ≥45%, which is ≥3% (absolute) higher than the baseline hematocrit or hematocrit ≥48%. Confirmation is defined as 2 consecutive hematocrit assessments that are ≥45% and at least 3% higher than the baseline hematocrit.
Time frame: Week 20 through Week 32
Change From Baseline in Hematocrit Over Time
Time frame: From Baseline to Week 32
Percentage of Participants Maintaining Hematocrit <45% During Week 0 to Week 32 and Week 0 to Week 52
Time frame: From Baseline to Weeks 32 and 52
Median Time to First Hematocrit ≥45% After Enrollment
Time frame: From Baseline to Week 52
Percentage of Participants With at Least One Hematocrit ≥48% During Week 0 to Week 32
Time frame: From Baseline to Week 32
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
An Adverse Event (AE) is any untoward medical occurrence in a clinical trial participant, temporally associated with the use of the trial intervention, whether or not the occurrence is considered related to the trial intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of the trial intervention. A TEAE is defined as an AE that commences on or after the first dose of the trial intervention and within 4 weeks after the last dose of the trial intervention.
Time frame: From Baseline to Week 56
Percentage of Participants With Adverse Events of Special Interest (AESIs)
An AE is any untoward medical occurrence in a clinical trial participant, temporally associated with the use of the trial intervention, whether or not the occurrence is considered related to the trial intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of the trial intervention. AESIs for this trial include cancer events.
Time frame: From Baseline to Week 56
Plasma Concentration-Time Data for Rusfertide
Time frame: From Baseline to Week 52
Number of Participants With Anti-Rusfertide Antibodies
Time frame: From Baseline to Week 56
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