Health Condition 1: D45- Polycythemia vera
Conditions
Interventions
Intervention1: PTG-300: Part 1 Dose Finding Phase: 28 weeks
all subjects will receive PTG-300.
Part 2 Blinded Randomized Withdrawal Phase: Up to 12 weeks, subjects are randomized 1:1 to continue PTG
Sponsors
Protagonist Therapeutics Inc
Eligibility
Inclusion criteria
Inclusion criteria: 1. Male and female subjects aged 18 to 85 years. 2. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of PV. 3. Hematocrit 4. Phlebotomy requiring defined as: a. Regularly spaced phlebotomies over at least 28 weeks prior to dosing. b. At least 3 phlebotomies during the 28 weeks prior to dosing. c. Last phlebotomy within approximately 12 weeks before Screening. 5. Records of all phlebotomies performed for at least 28 weeks (and preferably up to 52 weeks) before dosing. 6. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2. 7. Women of childbearing potential and men agree to use medically acceptable contraception ( 8. Subject understands the study procedures, is willing and able to adhere to study requirements and agrees to participate in the study by giving written informed consent. 9. 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 AEs due to cytoreductive therapy. 10. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib must have received cytoreductive therapy for at least 24 weeks and be on a stable dose or have a decreasing dose (Medical Monitor approval required) for at least 8 weeks before dosing with no planned change in dose.
Exclusion criteria
Exclusion criteria: 1. Clinically meaningful laboratory abnormalities at Screening including, but not limited to: a. Absolute neutrophil count lesser than 1000/Ã?¼L b. Platelet count lesser than 100,000/Ã?¼L c. Estimated glomerular filtration rate (eGFR) lesser than 40 mL/min/1.73 m2 d. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) equal or greater than 2.5 Ã?â?? upper limit of normal (ULN) or direct bilirubin greater than 1.5 Ã?â?? ULN. 2. Pregnant or lactating females. 3. Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 1 month of Screening. 4. Active or chronic bleeding within 4 weeks of Screening. 5. Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT). 6. Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 3 months of dosing; any infection requiring antimicrobial therapy within 4 weeks of dosing. Prophylactic antibodies are allowed. 7. 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. 8. Known primary or secondary immunodeficiency. 9. Positive for hepatitis B or hepatitis C or known human immunodeficiency virus (HIV) infection. 10. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study. 11. History of invasive malignancies within the last 2 years, except non-melanoma skin cancer and localized cured prostate cancer, cervical cancer, and ductal carcinoma in situ (DCIS). 12. Current or recent history of alcohol dependence or illicit drug use within 1 year prior to Screening. 13. Subject is unable to give informed consent. 14. Receipt of an investigational agent within 30 days of Screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Estimate PTG-300ââ?¬â?¢s efficacy in phlebotomy-requiring polycythemia vera (PV) subjects including changes in phlebotomy requirement and splenomegaly. 2. Assess the safety of PTG-300 in PV subjects who are phlebotomy requiring.Timepoint: 1. The study will consist of a Screening period (Days -28 to -1). Part 1- Dose Finding Phase (Open Label Treatment) Part 2- Blinded Withdrawal Phase (Randomized Treatment) Part 3- OLE Phase (Open Label Extension) 2. Safety will be assessed throughout the study conduct. Safety labs, physical examination (including vitals), and ECG will be performed for the same every 4 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Examine the effect of PTG-300 on the modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) diary. 2.Examine the effect of PTG-300 on markers of iron metabolism. Timepoint: 1. In Part 1, Part 2, Part 3 & EOT study visits. 2. In Part 1, Part 2, Part 3 study & EOT study visits. | — |
Countries
India, United States of America
Contacts
Public ContactDr Jayashri Krishnan
JSS Medical Research Asia Pacific Private Limited
Outcome results
None listed