VERIFY: A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

Sponsor
Protagonist Therapeutics, Inc. (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05210790
Collaborator
(none)
250
35
2
38
7.1
0.2

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

Study Type:
Interventional
Anticipated Enrollment :
250 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
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.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.
Masking:
Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients With Polycythemia Vera
Actual Study Start Date :
Apr 1, 2022
Anticipated Primary Completion Date :
Feb 1, 2024
Anticipated Study Completion Date :
Jun 1, 2025

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

  1. 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

  1. Comparison of mean number of phlebotomies between rusfertide and placebo. [Week 0 to Week 32]

  2. Proportion of subjects with HCT values <45% for rusfertide and placebo. [Week 0 to Week 32]

  3. Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo. [Week 32]

  4. Comparison of mean change from baseline in total MFSAF total score. [Week 32]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No

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.

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.
Responsible Party:
Protagonist Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT05210790
Other Study ID Numbers:
  • PTG-300-11
First Posted:
Jan 27, 2022
Last Update Posted:
Aug 1, 2022
Last Verified:
Jul 1, 2022
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Protagonist Therapeutics, Inc.
Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 1, 2022