A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

Phase 3

Active, not recruiting

• Conditions: Polycythemia Vera
• Interventions: Drug: Placebo; Drug: Rusfertide

• Registration Number: NCT05210790
• Lead Sponsor: Protagonist Therapeutics, Inc.

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.

Study Timeline

• Study First Submitted: 2022-01-14
• Study First Submitted QC: 2022-01-14
• Study First Posted: 2022-01-27
• Study Start: 2022-04-01
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-15
• Last Update Posted: 2024-07-17
• Primary Completion: 2025-02
• Study Completion: 2025-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 250

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Rusfertide	Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)
Placebo	Placebo	Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)
Rusfertide	Rusfertide	Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Proportion of subjects with HCT values <45% for rusfertide and placebo.	Week 0 to Week 32
Comparison of mean number of phlebotomies between 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

Trial Locations

Locations (171)

Infirmary Cancer Care; 🇺🇸 Mobile, Alabama, United States

Palo Verde Hematology-Oncology; 🇺🇸 Glendale, Arizona, United States

City of Hope Medical Center, Duarte; 🇺🇸 Duarte, California, United States

California Cancer Associates for Research and Excellence - Fresno; 🇺🇸 Fresno, California, United States

Marin Cancer Care; 🇺🇸 Greenbrae, California, United States

City of Hope Medical Center, Lenner; 🇺🇸 Irvine, California, United States

University of California, San Diego (UCSD) - Moores Cancer Center; 🇺🇸 La Jolla, California, United States

University of California, Los Angeles (UCLA) - Medical Center; 🇺🇸 Los Angeles, California, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

Pomona Valley Hospital Medical Center; 🇺🇸 Pomona, California, United States

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