A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

Overview

The study is designed to evaluate the safety and efficacy of rusfertide in subjects with polycythemia vera (PV) in maintaining hematocrit control and in improving symptoms of PV.

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.

Eligibility

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.