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ACTIVE NOT RECRUITING

NCT05210790

PHASE3

A Study of Rusfertide in Adults With Polycythemia Vera (PV)

Sponsor: Protagonist Therapeutics, Inc.

View on ClinicalTrials.gov

Summary

Polycythemia vera (PV) is a long-term condition in which the bone marrow makes too many red blood cells (RBCs). Bone marrow is the soft tissue inside the large bones where blood cells are made. When there are too many RBCs, called erythrocytosis, the blood can become thicker and move less easily through blood vessels and organs. This can raise the risk of blood clots (thrombosis) in veins or arteries. Sometimes, these clots can be life-threatening and may cause a stroke or heart attack. Treatment aims to keep the percentage of RBCs in the blood (hematocrit) in a safe range. For adults with PV, this means keeping the hematocrit below 45%. This helps lower the blood thickness and the risk of thrombosis. PV can also be linked to low iron levels (iron deficiency), because the body uses iron to make more RBCs. Many people with PV are treated with blood removal (phlebotomy) to lower RBC levels in the blood. This can worsen the iron deficiency, because each blood removal also takes iron out of the body. Rusfertide is a medicine that lowers the amount of iron available in the blood. It works like hepcidin, a natural hormone that helps control iron levels in the body. By limiting iron available for RBC production, rusfertide may help keep hematocrit below 45% and may improve symptoms. It may also reduce, or even remove, the need for phlebotomy in people. The main aim of this study is to check how well rusfertide works compared with placebo to lower the number of phlebotomies. A placebo looks like rusfertide but does not have any medicine in it. Other aims are to learn how safe rusfertide is compared with placebo and to understand how well it works to reduce the need for phlebotomy and to keep hematocrit levels under control. The study also wants to learn how rusfertide moves through the body (pharmacokinetics) and how it affects the body (pharmacodynamics or PD). This study has 4 parts that follow each other: * In part 1a, participants will receive either rusfertide or placebo. * In part 1b, all participants will receive rusfertide. * In part 2, participants can continue to receive rusfertide if they benefit from treatment with rusfertide. * Part 3 will include participants who finish Part 2 and still benefit from rusfertide. During the study, participants will visit the study clinical several times at fixed timepoints.

Official title: A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300; TAK-121) in Patients With Polycythemia Vera

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

293

Start Date

2022-04-11

Completion Date

2028-08-31

Last Updated

2026-10-19

Healthy Volunteers

Conditions

Polycythemia Vera

Interventions

DRUG

Rusfertide

Rusfertide SC injection

DRUG

Placebo

Rusfertide-matching placebo SC injection

Inclusion criteria: 1. Male and female participants aged 18 (or the minimum country specific age of consent if greater than \[\>\]18) years or older. 2. Participant understands the study procedures, is willing and able to adhere to study requirements and agrees to participate in the study by giving written informed consent. 3. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of PV. 4. Phlebotomy requiring defined as ALL of the following: 1. At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization, and 2. Last phlebotomy due to inadequate hematocrit control within 3 months before randomization, and 3. No phlebotomy within 6 days prior to randomization (do not include day of phlebotomy and day of randomization in the 6-day count). 5. Complete blood count (CBC) values immediately prior to randomization: 1. Hematocrit \<45%, 2. White blood cell count (WBC) 4000 cells per microlitre (/μL) to 20,000/μL (inclusive), and 3. Platelets 100,000/μL to 1,000,000/μL (inclusive). 6. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2. 7. Women of childbearing potential (WOCBP) agree to use medically acceptable contraception (\<1% annual failure rate) during the study and for 30 days after the last dose of study drug. 8. A female participant must agree not to donate eggs (ova, oocytes) for the purposes of assisted reproduction during the study and for a period of 30 days after receiving the last dose of study medication. 9. Men with partners of childbearing potential agree to use medically acceptable contraception (\<1% annual failure rate) during the study and for 90 days after the last dose of study drug. In addition, men must use a condom during the study and for 90 days after the last dose of study drug regardless of the partner's childbearing potential. 10. A male participant must agree not to donate sperm for the purpose of reproduction during the study and for a minimum of 90 days after receiving the last dose. 11. Participants receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen as follows: 1. Hydroxyurea - at least 2 months 2. Janus kinase (JAK) inhibitor - at least 2 months 3. Interferon - at least 6 months. 12. Participants treated with phlebotomy alone at randomization must have stopped: 1. Hydroxyurea at least 2 months before screening 2. JAK inhibitor at least 2 months before screening 3. Interferon at least 6 months before screening. Exclusion criteria: 1. Clinically meaningful laboratory abnormalities at Screening, including but not limited to the following: 1. Estimated glomerular filtration rate (eGFR) \<15 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) 2. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5×upper limit of normal (ULN) 3. Total bilirubin \>1.5×ULN 2. Participants who required phlebotomy at HCT levels lower than 45%. 3. Pregnant or lactating females. 4. Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization. 5. Active or chronic bleeding within 2 months prior to randomization. 6. Met the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment. 7. Any infection requiring systemic therapy within 1 month of dosing except controlled human immunodeficiency virus (HIV), hepatitis B, and hepatitis C. Prophylactic therapies were allowed. 8. Any serious or unstable medical condition (e.g., poorly controlled HIV infection) or uncontrolled psychiatric condition as judged by the Investigator that would impair the participant's ability to participate in the study. 9. Major surgical procedure within 2 months prior to randomization unless the participant had fully recovered from surgery or planned major elective surgery during the study. 10. History of invasive malignancies within the last 5 years, except 1. localized cured cancer (e.g., prostate cancer and cervical cancer) 2. localized cured in situ or Stage 1 squamous cell carcinoma (SCC) of the skin, basal cell carcinoma (BCC) of the skin, or in situ melanoma of the skin. 11. Participants with in situ or Stage 1 SCC of the skin, in situ or Stage 1 BCC of the skin, or in situ melanoma of the skin identified during the required dermatology examination at Screening unless the cancer was adequately treated (i.e., treatment that was expected to be curative, such as Mohs surgery) before randomization. Note: Suspicious lesions were to be biopsied and results made available before randomization. 12. Participants with active alcohol or drug addiction that would have interfered with their ability to comply with study requirements. 13. Participants who did not complete at least 4 days of MFSAF assessments within 1 week prior to randomization. 14. Receipt of an investigational agent within 2 months or 5 half-lives, whichever was longer, prior to randomization. 15. Received busulfan, pipobroman, or 32Phosphorus within 7 months prior to Screening. 16. Participants with hypersensitivity to rusfertide or to any of the excipients or placebo. 17. Participants with any lesion or mass detected by physical examination or imaging during screening that was suspicious for malignancy unless evaluated and assessed to be not malignant.

Locations (184)

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

Sharp Memorial Hospital

San Diego, California, United States

University of California, San Francisco

San Francisco, California, United States

City of Hope Medical Center, Upland

Upland, California, United States

Rocky Mountain Cancer Centers

Aurora, Colorado, United States

University of Colorado - Aurora Cancer Center

Aurora, Colorado, United States

Rocky Mountain Cancer Centers - Boulder

Boulder, Colorado, United States

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, United States

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, United States

Rocky Mountain Cancer Centers

Denver, Colorado, United States

Rocky Mountain Cancer Centers

Lakewood, Colorado, United States

Rocky Mountain Cancer Centers

Littleton, Colorado, United States

Rocky Mountain Cancer Centers

Lone Tree, Colorado, United States

Yale University School of Medicine - Yale Cancer Center

New Haven, Connecticut, United States

BRCR Medical Center

Plantation, Florida, United States

BayCare Suncoast Medical Clinic - Hematology/Oncology

St. Petersburg, Florida, United States

Moffitt Cancer Center

Tampa, Florida, United States

Northwestern Memorial Hospital

Chicago, Illinois, United States

Northwestern Medicine Lake Forest Hospital

Lake Forest, Illinois, United States

Northwest Oncology and Hematology

Rolling Meadows, Illinois, United States

Orchard Healthcare Research Inc

Skokie, Illinois, United States

Fort Wayne Medical Oncology and Hematology

Fort Wayne, Indiana, United States

Mission Cancer & Blood

Des Moines, Iowa, United States

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

University of Kansas Cancer Center

Kansas City, Kansas, United States

Pontchartrain Cancer Center

Covington, Louisiana, United States

Pontchartrain Cancer Center

Hammond, Louisiana, United States

American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)

Bethesda, Maryland, United States

University of Michigan

Ann Arbor, Michigan, United States

Cancer Care Specialists

Reno, Nevada, United States

Memorial Sloan Kettering Cancer Center - BASKING RIDGE

Basking Ridge, New Jersey, United States

Monmouth Medical Center Southern Campus

Lakewood, New Jersey, United States

Monmouth Medical Center

Long Branch, New Jersey, United States

Memorial Sloan Kettering Cancer Center - MONMOUTH

Middletown, New Jersey, United States

Memorial Sloan Kettering Cancer Center - BERGEN

Montvale, New Jersey, United States

Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey, United States

New York Cancer and Blood Specialists

Babylon, New York, United States

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States

Memorial Sloan Kettering Cancer Center - COMMACK

Commack, New York, United States

Memorial Sloan Kettering Cancer Center - WESTCHESTER

Harrison, New York, United States

New York Cancer and Blood Specialists

New Hyde Park, New York, United States

Memorial Sloan Kettering Cancer Center - KOCH

New York, New York, United States

New York Cancer and Blood Specialists

New York, New York, United States

Ichan School of Medicine at Mount Sinai

New York, New York, United States

New York Presbyterian Hospital/Weill Cornell Medical Center

New York, New York, United States

New York Cancer and Blood Specialists

Patchogue, New York, United States

New York Cancer and Blood Specialists

Port Jefferson Station, New York, United States

New York Cancer and Blood Specialists

Riverhead, New York, United States

Wilmot Cancer Institute, University of Rochester

Rochester, New York, United States

Montefiore Einstein Center for Cancer Care

The Bronx, New York, United States

Montefiore Medical Center

The Bronx, New York, United States

New York Cancer and Blood Specialists

The Bronx, New York, United States

Memorial Sloan Kettering Cancer Center - NASSAU

Uniondale, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

East Carolina University

Greenville, North Carolina, United States

Gabrail Cancer Center

Canton, Ohio, United States

TriHealth Cancer Institute - Good Samaritan Hospital

Cincinnati, Ohio, United States

Good Samaritan Infusion Center - Kenwood

Cincinnati, Ohio, United States

Good Samaritan Infusion Center - Thomas

Cincinnati, Ohio, United States

The Ohio State University

Columbus, Ohio, United States

Ohio Health Research Institute

Columbus, Ohio, United States

Oncology Associates of Oregon

Eugene, Oregon, United States

Oregon Health and Science University (OHSU) Knight Cancer Institute

Portland, Oregon, United States

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

MD Anderson Cancer Center

Houston, Texas, United States

Texas Oncology San Antonio

San Antonio, Texas, United States

Community Cancer Trials of Utah

Ogden, Utah, United States

University of Washington - Fred Hutchinson Cancer Center

Seattle, Washington, United States

HSHS St. Vincent Hospital

Green Bay, Wisconsin, United States

HSHS St. Mary's Hospital

Green Bay, Wisconsin, United States

One Clinical Research

Nedlands, Perth, Australia

Royal Adelaide Hospital

Adelaide, South Australia, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

Medizinische Universitaet Graz

Graz, Austria

Kepler Universitaets Klinikum GmbH

Linz, Austria

Krankenhaus der Elisabethinen Linz

Linz, Austria

Medizinische Universitaet Wien

Vienna, Austria

Wiener Gesundheitsverbund-Klinik Hietzing 5-Med.

Vienna, Austria

Klinikum Wels-Grieskirchen Gmb

Wels, Austria

ZNA Stuivenberg

Antwerp, Belgium

University Hospital Gent

Ghent, Belgium

Campus Gasthuisberg

Leuven, Belgium

The University of Alberta - Edmonton

Edmonton, Alberta, Canada

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Princess Margaret Hospital

Toronto, Ontario, Canada

Jewish General Hospital

Montreal, Quebec, Canada

Centro Medico Inmunocel

Las Condes, Santiago Metropolitan, Chile

Centro de Estudios Clinicos SAGA

Providencia, Santiago Metropolitan, Chile

Biocenter Centro Clinico de Investigacion

Concepción, Chile

Fakultni nemocnice Brno

Brno, Czechia

And 84 more locations on ClinicalTrials.gov

Clinical Research Directory

A Study of Rusfertide in Adults With Polycythemia Vera (PV)

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (184)