A Study of Rusfertide in Japanese Adults With Polycythemia Vera
ConditionPolycythemia Vera
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorTakeda
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
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
Disqualifiers
eGFR <15 mL/min/1.73 m² 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%.
Trial design
Design model
Single group
Treatments tested in this trial
Rusfertide
DrugRusfertide injections administered subcutaneously.
Treatment groups
9 Participants
are divided into 1 treatment groupGroup A: RusfertideExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Percentage of Participants Achieving a Response Starting at Week 20 through Week 32
Response is defined as absence of phlebotomy eligibility. Phlebotomy eligibility is defined as either: A confirmed hematocrit 45% or more and that is at least 3% (absolute) higher than the baseline hematocrit. Confirmation is defined as 2 consecutive hematocrit assessments that are 45% or more and at least 3% higher than the baseline hematocrit (if it is observed at Week 32, confirmation is not required). OR a hematocrit 48% or more.
Time frame
Week 20, up to Week 32Secondary outcomes
1
Mean Number of Phlebotomies through Week 32
Time frame
Baseline, up to Week 322
Percentage of Participants with All Hematocrit Values Less Than 45%
A single hematocrit value \>=45% is allowed.
Time frame
Baseline, up to Week 323
Median Time to First Hematocrit Value 45% Or More After Enrollment
Time frame
Baseline, up to Week 324
Percentage of Participants with At Least One Hematocrit Value 48% Or More
Time frame
Baseline, up to Week 32 Source ClinicalTrials.gov