A Phase 2 Open-label Trial to Evaluate the Efficacy and Safety of Rusfertide in Japanese Patients With Polycythemia Vera
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.
• 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
‣ 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).
• Note: Phlebotomies performed within an 8-day period will be counted as a single phlebotomy.
• Hematology test values at screening:
∙ Hematocrit \<45%
‣ WBC 4,000/µL to 20,000/µL (inclusive), and
‣ Platelets 100,000/µL to 1,000,000/µL (inclusive).
• ECOG performance status 0, 1 or 2.
• WOCBP agree to use at least 1 form of highly effective contraception during the trial and for 30 days after the last dose of trial intervention.
• A female participant must agree not to donate eggs (ova, oocytes) for the purposes of assisted reproduction during the trial and for a period of 30 days after receiving the last dose of trial medication.
• A fertile man agree to use a condom, preferably combined with at least 1 form of acceptable contraception for any WOCBP partner(s) during the trial and for 90 days after the last dose of trial intervention.
⁃ A male participant must agree not to donate sperm for the purpose of reproduction during the trial and for a minimum of 90 days after receiving the last dose.
⁃ Participants receiving CRT at trial intervention must be on a stable PV therapy regimen as follows:
• Hydroxyurea - at least 8 weeks
∙ JAK inhibitor - at least 8 weeks
∙ Interferon - at least 24 weeks. Note: A stable dose regimen of CRT does not mean an unchanged dose regimen. Temporary adjustments in dose regimen or temporary suspension of dosing are allowed. However, the total weekly dose of hydroxyurea and JAK inhibitor or total monthly dose of interferon may not be higher at trial intervention than the dose at the beginning of the pre-trial intervention observation period. The pre-trial intervention observation period is 8 weeks for hydroxyurea and JAK inhibitor and 24 weeks for interferon.
⁃ Participants treated with phlebotomy alone at trial intervention must have stopped:
• Hydroxyurea at least 8 weeks before trial intervention
∙ JAK inhibitor at least 8 weeks before trial intervention
∙ Interferon at least 24 weeks before trial intervention.