A Phase 3 Study of Rusfertide (PTG-300) in Patients with Polycythemia Vera
Research type
Research Study
Full title
A Phase 3 Study of Rusfertide (PTG-300) in Patients with Polycythemia Vera
IRAS ID
1005184
Contact name
Ciro Rinaldi
Contact email
Sponsor organisation
Protagonist Therapeutics, Inc.
Eudract number
2021-004732-29
Clinicaltrials.gov Identifier
Research summary
Polycythaemia Vera (PV) is a blood disease in which patients make too many red blood cells. Patients are at increased risk of blood clots, strokes and heart attacks because they have too many red blood cells. Treatment is designed to maintain red blood cells at a safe level. Haematocrit is the routine blood test used to monitor the number of red blood cells and control of Polycythaemia Vera.
The purpose of this study is to determine whether the study medicine, rusfertide is safe and effective for treating Polycythaemia Vera. Rusfertide (also known as PTG-300) is an experimental study medicine that lowers the level of iron in the blood. Iron is required for production of red blood cells. Rusfertide has been tested for safety and efficacy in small clinical trials in patients with Polycythaemia Vera, a blood disorder called beta-thalassemia, and in a disorder of iron metabolism called hereditary hemochromatosis.
This is a Phase 3 study in subjects with polycythemia vera.
• Part 1a: randomized, double-blind, placebo-controlled, add-on parallel-group period for 32 weeks. Subjects will be randomized 1:1 in a blinded fashion to 32 weeks of add-on rusfertide or placebo treatment. Rusfertide or placebo will be added-on to each subject’s ongoing therapy for PV. Randomization will be stratified by ongoing PV therapy.
• Part 1b: open-label treatment phase during which all subjects who complete Part 1a successfully will receive rusfertide for 20 weeks (Week 32 through Week 52).
• Part 2: long term extension for 104 weeks during which all subjects who complete Part 1b will continue to receive rusfertide.
Approximately 250 participants will take part in this study at approximately 100 study sites globally. Enrollment will be competitive, thus there won’t be a cap at the sites.REC name
East of England - Cambridge East Research Ethics Committee
REC reference
22/EE/0157
Date of REC Opinion
5 Sep 2022
REC opinion
Further Information Favourable Opinion