Safety Evaluation Study for Patients With Polycythemia Vera

Study Overview
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Brief Summary
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This is PhaseI Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of PPMX-T003 in Polycythemia Vera

Official Title
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Phase I, Open-Label, Multicentre, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of A Single Intravenous PPMX-T003 in Polycythemia Vera

Conditions
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Polycythemia Vera

Intervention / Treatment
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* Drug: PPMX-T003


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed diagnosis of PV according to either the 2008 or 2016 WHO classification criteria PV patients being only treated with phlebotomy and the interval is 4-9 weeks Exclusion Criteria: Patients administrated drugs for PV treatment such as hydroxyurea or ruxolitinib (aspirin is excluded)

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PPMX-T003<br>This drug should be administered within 48 hours after the phlebotomy. In addition, as a dose escalation design, 4 doses of 0.25 mg/kg, 0.4 mg/kg, 0.64 mg/kg, and 1 mg/kg are administered to the same subject, when the next phlebotomy required during observation period after the 1st administration. | Drug: PPMX-T003<br>* As an observation and evaluation method, for each dose, in principle, inpatient observation is performed for 1 week from the day before administration, and then safety and pharmacodynamic tests are evaluated by visiting the hospital every 2 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study |  | 45 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Observed maximum plasma concentration [Cmax] | Day1,Day2,Day7 | 
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Terminal elimination rate constant [λz] | Day1,Day2,Day7 | 
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Area under the concentration-time curve from pre-dose [time 0] to the time of the last quantifiable concentration [AUC0-t] | Day1,Day2,Day7 | 
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Area under the concentration-time curve from pre-dose [time 0] extrapolated to infinite time [AUC0-inf] | Day1,Day2,Day7 | 
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Apparent terminal half-life [t½] | Day1,Day2,Day7 | 
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Apparent systemic clearance [CL] | Day1,Day2,Day7 | 
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Volume of distribution [Vd] | Day1,Day2,Day7 | 
| Examining the expression rate of anti-drug antibodies (ADA) |  | Day1,Day21,up to 45weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Myeloproliferative neoplasms