Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

NCT ID: NCT04057040

Last Updated: 2025-08-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-01

Study Completion Date

2024-06-17

Brief Summary

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This is a Phase 2 study with an open-label dose escalation phase followed by a blinded withdrawal phase and an open label extension. The study is designed to monitor the PTG-300 safety profile and to obtain preliminary evidence of efficacy of PTG-300 for the treatment of phlebotomy-requiring polycythemia vera.

Detailed Description

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Phase 2 study in approximately sixty subjects previously diagnosed with Polycythemia Vera who require phlebotomy on a routine basis. There is a 28 week dose finding phase to identify a dose that maintains hematocrit \<45%. Subjects who successfully complete the dose finding phase will be entered into a 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into an up to 3 year open label extension to investigate long term safety.

Conditions

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Polycythemia Vera

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Part 1:

28 week open-label dose escalation phase in which each subject's dose of PTG-300 is titrated to achieve a hematocrit \<45%.

Part 2:

12-week blinded randomized withdrawal phase. Subjects are randomized 1:1 to continue PTG-300 or to receive placebo.

Part 3:

Up to 3 year open label extension.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
Part 1 open label, Part 2 blinded, Part 3 open label

Study Groups

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Dose finding PTG-300 (Part 1); PTG-300 (Part 2); Open label extension PTG-300 (Part 3)

Group Type EXPERIMENTAL

PTG-300

Intervention Type DRUG

Active

Dose finding PTG-300 (Part 1); Placebo (Part 2); Open label extension PTG-300 (Part 3)

Group Type EXPERIMENTAL

PTG-300

Intervention Type DRUG

Active

Placebo

Intervention Type DRUG

Placebo

Interventions

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PTG-300

Active

Intervention Type DRUG

Placebo

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Male and female subjects aged 18 years or older.
2. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera.
3. Records of all phlebotomies performed for at least 28 weeks (preferably up to 52 weeks) before dosing are available.
4. Subjects who are not receiving cytoreductive therapy must have been discontinued from any prior cytoreductive therapy for at least 24 weeks before screening and have recovered from any adverse events due to cytoreductive therapy.
5. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib must have received cytoreductive therapy for at least 24 weeks and be on a stable dose or have a decreasing dose (Medical Monitor approval required) for at least 8 weeks before dosing and with no planned change in dose.

Exclusion Criteria

1. Active or chronic bleeding within 4 weeks of screening.
2. Meets the criteria for post-PCV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).
3. Known primary or secondary immunodeficiency.
4. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Protagonist Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic - Mayo Clinic Hospital

Phoenix, Arizona, United States

Site Status

Marin Cancer Care

Greenbrae, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

Moffitt Cancer Center

Tampa, Florida, United States

Site Status

University of Kansas

Westwood, Kansas, United States

Site Status

Pontchartrain Cancer Care

Covington, Louisiana, United States

Site Status

Center for Cancer and Blood Disorders

Bethesda, Maryland, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Karmanos Cancer Center

Detroit, Michigan, United States

Site Status

Mount Sinai

New York, New York, United States

Site Status

New York Presbyterian Hospital - Weill Cornell Medical Center

New York, New York, United States

Site Status

Cleveland Clinic - Taussig Cancer Center

Cleveland, Ohio, United States

Site Status

Mary Crowley Cancer Research Center

Dallas, Texas, United States

Site Status

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Sahyadri Super Specialty Hospital

Pune, Maharashtra, India

Site Status

All India Institute of Medical Sciences

Rishikesh, Uttarakhand, India

Site Status

Countries

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United States India

References

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Kremyanskaya M, Kuykendall AT, Pemmaraju N, Ritchie EK, Gotlib J, Gerds A, Palmer J, Pettit K, Nath UK, Yacoub A, Molina A, Saks SR, Modi NB, Valone FH, Khanna S, Gupta S, Verstovsek S, Ginzburg YZ, Hoffman R; REVIVE Trial Investigators. Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera. N Engl J Med. 2024 Feb 22;390(8):723-735. doi: 10.1056/NEJMoa2308809.

Reference Type DERIVED
PMID: 38381675 (View on PubMed)

Other Identifiers

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PTG-300-04

Identifier Type: -

Identifier Source: org_study_id

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