VERIFY: A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera
Study Details
Study Description
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.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 3 |
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.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: Rusfertide Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label) |
Drug: Rusfertide
Experimental drug
|
Experimental: Placebo Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label) |
Drug: Placebo
Placebo
Drug: Rusfertide
Experimental drug
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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]
Eligibility Criteria
Criteria
Main Inclusion Criteria: All subjects must meet ALL of the following inclusion criteria to be enrolled. There are additional inclusion criteria.
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Male and female subjects aged 18 (or the country specific minimum age of consent >18) years or older.
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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.
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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.
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CBC values immediately prior to randomization:
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Hematocrit <45%,
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WBC 4000/μL to 20,000/μL (inclusive), and
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Platelets 100,000/μL to 1,000,000/μL (inclusive)
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Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen.
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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.
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Clinically meaningful laboratory abnormalities at Screening.
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Subjects who require phlebotomy at hematocrit levels lower than 45%.
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Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.
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Active or chronic bleeding within 2 months prior to randomization.
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History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer.
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Subjects with non-invasive non-melanomatous (e.g., squamous cell or basal cell carcinoma) skin cancer during screening unless adequately treated before randomization.
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Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | California Cancer Associates for Research and Excellence - Fresno | Fresno | California | United States | 93720 |
2 | Marin Cancer Care | Greenbrae | California | United States | 94904 |
3 | University of California, San Diego (UCSD) - Moores Cancer Center | La Jolla | California | United States | 92037 |
4 | University of California, Los Angeles (UCLA) - Medical Center | Los Angeles | California | United States | 90095 |
5 | Stanford University | Palo Alto | California | United States | 94304 |
6 | BRCR Medical Center | Plantation | Florida | United States | 33322 |
7 | Moffitt Cancer Center | Tampa | Florida | United States | 33612 |
8 | Northwest Oncology and Hematology | Rolling Meadows | Illinois | United States | 60008 |
9 | Orchard Healthcare Research Inc | Skokie | Illinois | United States | 60046 |
10 | University of Kansas Cancer Center | Kansas City | Kansas | United States | 66160 |
11 | American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders) | Bethesda | Maryland | United States | 20817 |
12 | University of Michigan | Ann Arbor | Michigan | United States | 48109 |
13 | Cancer Care Specialists | Reno | Nevada | United States | 89511 |
14 | Duke University Medical Center | Durham | North Carolina | United States | 27710 |
15 | Gabrail Cancer Center | Canton | Ohio | United States | 44718 |
16 | MD Anderson Cancer Center | Houston | Texas | United States | 77030 |
17 | One Clinical Research | Nedlands | Perth | Australia | |
18 | Royal Adelaide Hospital | Adelaide | South Australia | Australia | 5000 |
19 | The Alfred Hospital | Melbourne | Victoria | Australia | 3004 |
20 | Princess Margaret Hospital | Toronto | Ontario | Canada | M5G 2C4 |
21 | Fakultni nemocnice Brno | Brno | Czechia | 625 00 | |
22 | Fakultni nemocnice Olomouc | Olomouc | Czechia | 779 00 | |
23 | Vseobecna fakultni nemocnice v Praze | Prague | Czechia | 128 08 | |
24 | AP-HP Hopital Saint-Louis | Paris | France | 75010 | |
25 | Uniklinik RWTH Aachen - Klinik fuer Haematologie, Onkologie, Haemostaseologie und Stammzelltransplantation (Med. Klinik IV) | Aachen | Germany | 52074 | |
26 | Universitaetsmedizin Greifswald | Greifswald | Germany | 17489 | |
27 | Queen Mary Hospital | Hong Kong | Hong Kong | ||
28 | Semmelweis Egyetem | Budapest | Hungary | 1085 | |
29 | Debreceni Egyetem Klinikai Kozpont | Debrecen | Hungary | 4032 | |
30 | Shaare Zedek Medical Center | Jerusalem | Israel | 9103102 | |
31 | Meir Medical Center | Kfar Saba | Israel | 4428164 | |
32 | Hospital Clinic Barcelona | Barcelona | Spain | 08036 | |
33 | Hospital Germans Trias i Pujol | Barcelona | Spain | 08916 | |
34 | Hospital General Universitario Gregorio Maranon | Madrid | Spain | 28007 | |
35 | Pilgrim Hospital | Boston | United Kingdom | PE21 9QS |
Sponsors and Collaborators
- Protagonist Therapeutics, Inc.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- PTG-300-11