A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More Inclusion & Exclusion 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT05210790
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Protagonist Therapeutics, Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	Australia, Austria, Belgium, Canada, Chile, Czechia, France, Germany, Hong Kong, Hungary, Israel, Italy, Mexico, Netherlands, Poland, Portugal, Spain, Turkey, United Kingdom, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Polycythemia Vera

Additional Details

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.

Arms & Interventions

Arms

Experimental: Rusfertide

Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)

Experimental: Placebo

Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)

Interventions

Drug: - Placebo

Placebo

Drug: - Rusfertide

Experimental drug

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Infirmary Cancer Care, Mobile, Alabama

Status

Recruiting

Address

Infirmary Cancer Care

Mobile, Alabama, 36607

Site Contact

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