Pfizer COVID-19 vaccine appointments are available to our patients. Sign up for Connect today to schedule your vaccination.

A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients with Polycythemia Vera

Clinical Trial Details

The purpose of this research study is to determine whether the study drug, rusfertide, is safe and effective for the treatment of polycythemia vera.

Rusfertide, also known as PTG-300, is an investigational study drug that lowers the level of iron in the blood. Rusfertide is considered investigational because it has not been approved by the United States Food and Drug Administration (FDA). So far, rusfertise has been tested for safety and efficacy in small clinical trials for people with polycythemia vera, those with a blood disorder called beta-thalassemia, and in a disorder of iron metabolism called hemochromatosis.

Rusfertide is given via an injection under the skin either in the abdomen or the thigh.

Participants in this study will be randomized into one of two groups to receive either the study drug or a placebo. Randomization is like flipping a coin and there is a 50/50 chance of being assigned to either group.

Study participants will also continue to receive their current treatment for polycythemia vera during the course of the study.

Participation will last up to 3 years and include 36 study visits, including 7 optional visits. Study visits will consist of physical exams, imaging, blood samples, and questionnaires.

Key Eligibility: 

   1. Males and females over the age of 18
   2. A diagnosis of polycythemia vera with either a JAK2 V617F mutation or JAK2 exon 12 mutation.
   3. Requires phlebotomy as treatment on a regular basis

Detailed eligibility will be reviewed when you contact the study team.

Study contact by location

Upper East Side - Manhattan


Niamh Savage

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:



Open to Enrollment

Age Group