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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