Polycythemia Vera
Conditions
Keywords
Polycythemia Vera, PV
Brief 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.
Detailed 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.
Interventions
DRUGPlacebo
Placebo
DRUGRusfertide
Experimental drug
Sponsors
Protagonist Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Intervention model description
Randomized, Double-blind (Part 1a): Subjects will be randomized in a blinded fashion to 32 weeks of rusfertide or placebo added-on to each subject's ongoing treatment for polycythemia vera. Open-label (Part 1b + Part 2): Open-label treatment phase during which all subjects who complete Part 1a successfully will receive rusfertide for 124 weeks.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion 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. * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of subjects achieving a response who receive rusfertide compared to placebo. | Week 20 through Week 32 | Response is defined as absence of phlebotomy eligibility. |
Secondary
| Measure | Time frame |
|---|---|
| Comparison of mean number of phlebotomies between rusfertide and placebo. | Week 0 to Week 32 |
| Proportion of subjects with HCT values <45% for rusfertide and placebo. | Week 0 to Week 32 |
| Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo. | Week 32 |
| Comparison of mean change from baseline in total MFSAF total score. | Week 32 |
Countries
Australia, Austria, Belgium, Canada, Chile, Czechia, France, Germany, Hong Kong, Hungary, Israel, Italy, Mexico, Netherlands, Poland, Portugal, Spain, Turkey (Türkiye), United Kingdom, United States
Outcome results
None listed