Hepcidin Mimetic in Patients With Polycythemia Vera
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.
- Polycythemia Vera
- Eligible Ages
- Over 18 Years
- Eligible Genders
- Accepts Healthy Volunteers
All subjects must meet ALL of the following inclusion criteria to be enrolled.
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 24 weeks (preferably up to 52 weeks) before screening 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 be on a stable dose for at least 24 weeks and be on a stable dose for at least 8 weeks before screening and with no planned change in dose.
Subjects must meet NONE of the following exclusion criteria to be enrolled:
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.
- Phase 2
- Study Type
- Intervention Model
- Sequential Assignment
- Intervention Model Description
- Part 1: 28 week open-label dose escalation phase in which each subject's dose of PTG-300 is increased at 4-week intervals until the subject reaches the maximum planned dose or has a pre-specified decrease in hematocrit from baseline. After a potentially clinically active dose is found, subjects will be maintained at that dose until Week 29. Part 2: 12-week blinded randomized withdrawal phase. Subjects are randomized 1:1 to continue PTG-300 or to receive placebo. Part 3: 1 year open label extension.
- Primary Purpose
- Double (Participant, Investigator)
- Masking Description
- Part 1 open label, Part 2 blinded, Part 3 open label.
Experimental Dose Escalation (Part 1)
|PTG-300 Subcutaneous (SC) Weekly Each subject's dose is increased at 4 week intervals until the subject reaches the maximum planned dose or has a prespecified decrease in hematocrit from baseline.||
Blinded Withdrawal (Part 2) PTG-300 or placebo
|PTG-300 or placebo Subcutaneous (SC) Weekly||
Experimental Open label extension (Part 3) PTG-300
|PTG-300 Subcutaneous (SC) Weekly||
- Protagonist Therapeutics
Study ContactStudy Director
Phase 2 study in approximately thirty subjects previously diagnosed with Polycythemia Vera who require phlebotomy on a routine basis. There is a 16 week dose finding phase followed by a dose stabilization phase. Subjects who successfully complete dose stabilization will be entered into 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into 1 year open label extension to investigate long term safety.