Description:
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.
Title
- Brief Title: Hepcidin Mimetic in Patients With Polycythemia Vera
- Official Title: A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera
Clinical Trial IDs
- ORG STUDY ID:
PTG-300-04
- NCT ID:
NCT04057040
Conditions
Interventions
Drug | Synonyms | Arms |
---|
PTG-300 | | Blinded Withdrawal (Part 2) PTG-300 or placebo |
Placebo | | Blinded Withdrawal (Part 2) PTG-300 or placebo |
Purpose
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.
Detailed Description
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 a 12 week randomized withdrawal phase to confirm the response. Subsequently
patients will enter into a 1 year open label extension to investigate long term safety.
Trial Arms
Name | Type | Description | Interventions |
---|
Experimental Dose Escalation (Part 1) | Experimental | PTG-300 Subcutaneous (SC) weekly. Each subject's PTG-300 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 | Experimental | PTG-300 or placebo Subcutaneous (SC) weekly. | |
Experimental Open label extension (Part 3) PTG-300 | Experimental | PTG-300 Subcutaneous (SC) weekly | |
Eligibility Criteria
Main Inclusion Criteria: 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.
Main Exclusion Criteria: 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.
Maximum Eligible Age: | N/A |
Minimum Eligible Age: | 18 Years |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Proportion of subjects with absence of phlebotomy eligibility |
Time Frame: | Week 42 |
Safety Issue: | |
Description: | "Phlebotomy eligibility" is defined as a confirmed hematocrit >45% that was ≥3% higher than baseline level or a confirmed hematocrit >48% |
Secondary Outcome Measures
Measure: | Change from baseline in serum iron |
Time Frame: | 42 weeks |
Safety Issue: | |
Description: | |
Measure: | Change from baseline in transferrin saturation (TSAT) |
Time Frame: | 42 weeks |
Safety Issue: | |
Description: | |
Measure: | Change from baseline in serum transferrin |
Time Frame: | 42 weeks |
Safety Issue: | |
Description: | |
Details
Phase: | Phase 2 |
Primary Purpose: | Interventional |
Overall Status: | Recruiting |
Lead Sponsor: | Protagonist Therapeutics, Inc. |
Trial Keywords
Last Updated
November 5, 2020