Safety Evaluation Study for Patients With Polycythemia Vera

Overview

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

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: August 1, 2023

Interventions

  • Drug: PPMX-T003
    • 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.

Arms, Groups and Cohorts

  • Experimental: PPMX-T003
    • 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.

Clinical Trial Outcome Measures

Primary Measures

  • Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study
    • Time Frame: 45 weeks

Secondary Measures

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

Participating in This Clinical Trial

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)

Gender Eligibility: All

Minimum Age: 20 Years

Maximum Age: 75 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Perseus Proteomics Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Contact(s)
    • Tadashi Matsuura, +81-3-5738-1705, tmatsuura@ppmx.com

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