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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05210790
Recruitment Status : Recruiting
First Posted : January 27, 2022
Last Update Posted : January 27, 2023
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.

Study Description
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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
Polycythemia Vera Drug: Placebo Drug: Rusfertide 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. Approximately 6 and 12 months after their last dose of rusfertide, subjects will have a post-study contact (e.g. by phone) for safety.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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.
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 : April 1, 2022
Estimated Primary Completion Date : February 2024
Estimated Study Completion Date : June 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Proportion of subjects achieving a response who receive rusfertide compared to placebo. [ Time Frame: 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. [ Time Frame: Week 0 to Week 32 ]
  2. Proportion of subjects with HCT values <45% for rusfertide and placebo. [ Time Frame: Week 0 to Week 32 ]
  3. Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo. [ Time Frame: Week 32 ]
  4. Comparison of mean change from baseline in total MFSAF total score. [ Time Frame: Week 32 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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 in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during screen unless the cancer is adequately treated before randomization.
  • Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05210790


Contacts
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Contact: Study Director 1-888-899-1543 ptgxclintrials@ptgx-inc.com

Locations
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Sponsors and Collaborators
Protagonist Therapeutics, Inc.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Protagonist Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05210790    
Other Study ID Numbers: PTG-300-11
First Posted: January 27, 2022    Key Record Dates
Last Update Posted: January 27, 2023
Last Verified: January 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Protagonist Therapeutics, Inc.:
Polycythemia Vera
PV
Additional relevant MeSH terms:
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Polycythemia Vera
Polycythemia
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
 Neoplasms by Site
Neoplasms
Bone Marrow Diseases
Myeloproliferative Disorders