Polycythemia Vera
Conditions
Keywords
PV
Brief summary
This is an open label, single arm trial of PTG-300 in subjects with PV who are newly diagnosed or for whom current therapy is not sufficient to control their hematocrit and have hematocrit \>48% prior to dosing. The PTG-300 dose and schedule may be adjusted every 2 to 4 weeks to maintain hematocrit \<45% with a target of \<43%. Subjects may receive PTG-300 treatment for up to 52 weeks.
Interventions
DRUGPTG-300
Hepcidin mimetic
Sponsors
Protagonist Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Known diagnosis of polycythemia vera. 2. Hematocrit \>48% before dosing. 3. Evidence of hematocrit \>48% three or more times in the 28 weeks before dosing or five or more times in 52 weeks before dosing (except for newly diagnosed patients). 4. Clinically reasonable alternative causes for erythrocytosis have been evaluated and excluded. 5. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2.
Exclusion criteria
1. Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 3 months of Screening. 2. Active or chronic bleeding within 4 weeks of Screening. 3. Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT). 4. Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 3 months of dosing; any infection requiring systemic antimicrobial therapy within 4 weeks of dosing. Prophylactic antibiotics are allowed. 5. Any serious or unstable medical or psychiatric condition that would prevent, (as judged by the Investigator) the subject from properly providing informed consent or any condition which would jeopardize compliance with the study. 6. Known primary or secondary immunodeficiency. 7. Known positive for active hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection. 8. Any surgical procedure requiring general anesthesia within 1 month prior to Screening or planned elective surgery during the study. 9. History of invasive malignancies within the last 2 years, except non-melanoma skin cancer and localized curatively treated prostate cancer or cervical cancer. 10. Current or recent history of alcohol dependence or illicit drug use within 1 year prior to Screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate PTG-300's efficacy in subjects with PV and baseline elevated hematocrit. | Estimate the proportion of subject with a hematocrit less than 45% at Week 16 | Proportion of subjects with hematocrit \<45% |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety of PTG-300 | Week 52 | Proportion of Subjects Treatment-Emergent Adverse Events |
Countries
Malaysia, South Korea
Outcome results
None listed