Clinical Trial Finder

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 and have either JAK2 V617F mutation or JAK2 exon 12 mutation.

• - 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 non-invasive non-melanomatous (e.g., squamous cell or basal cell carcinoma) skin cancer during screening unless adequately treated before randomization.

• - Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.

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.

Arms

Experimental: Rusfertide

Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)

Experimental: Placebo

Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)

Interventions

Drug: - Placebo

Placebo

Drug: - Rusfertide

Experimental drug