A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

• Status: Not yet recruiting
• Trial end date: 2025-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Protagonist Therapeutics, Inc.

Criteria

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.