Trial details

Trial short name: PTG 300 11

Official title: A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients with Polycythemia Vera (PTG 300 11)

Principal Investigator: Dr. James Kennedy

Cancer type: Blood Related
Cancer location: Blood Related
Disease stage: Early Cancer
Trial phase: Phase 3
Intervention: Rusfertide vs Placebo

Registration #: NCT05210790

Trial description:
Phase 3 study in approximately 250 subjects previously diagnosed with polycythemia vera (PV) who require phlebotomy on a routine basis. There is a 32-week period during which rusfertide or placebo will be added-on to each subject's ongoing therapy for polycythemia vera which may include phlebotomy only or phlebotomy plus stable doses of either of hydroxyurea, interferon and/or ruxolitinib. All subjects who successfully complete the double blind 32-week portion of the study will receive rusfertide for 124 weeks. Approximately 6 and 12 months after their last dose of rusfertide, subjects will have a post-study contact (e.g. by phone) for safety.

Main Inclusion Criteria: All subjects must meet ALL of the following inclusion criteria to be enrolled. There are additional inclusion criteria. • Male and female subjects aged 18 (or the country specific minimum age of consent >18) years or older. • Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera. • At least 3 phlebotomies due to inadequate hematocrit control in 6 months before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization. • CBC values immediately prior to randomization: 1.Hematocrit <45%, 2.WBC 4000/μL to 20,000/μL (inclusive), and 3.Platelets 100,000/μL to 1,000,000/μL (inclusive) • Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen. • Subjects treated with phlebotomy alone at randomization must have stopped cytoreductive therapy 2 to 6 months before screening. Main Exclusion Criteria: Subjects must meet NONE of the following exclusion criteria to be enrolled. There are additional exclusion criteria. • Clinically meaningful laboratory abnormalities at Screening. • Subjects who require phlebotomy at hematocrit levels lower than 45%. • Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization. • Active or chronic bleeding within 2 months prior to randomization. • History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer. • Subjects with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during screen unless the cancer is adequately treated before randomization. • Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.