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Safety Evaluation Study for Patients With Polycythemia Vera

Primary Purpose

Polycythemia Vera

Status
Recruiting
Phase
Phase 1
Locations
Japan
Study Type
Interventional
Intervention
PPMX-T003
Sponsored by
Perseus Proteomics Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycythemia Vera focused on measuring Myeloproliferative neoplasms

Eligibility Criteria

20 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • Kansai Medical University HospitalRecruiting
  • Shimane University HospitalRecruiting
  • Osaka City University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PPMX-T003

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study

Secondary Outcome Measures

Evaluation of pharmacokinetics of PPMX-T003 after single dose
Observed maximum plasma concentration [Cmax]
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Terminal elimination rate constant [λz]
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]
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]
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Apparent terminal half-life [t½]
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Apparent systemic clearance [CL]
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Volume of distribution [Vd]
Examining the expression rate of anti-drug antibodies (ADA)

Full Information

First Posted
September 14, 2021
Last Updated
July 21, 2023
Sponsor
Perseus Proteomics Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05074550
Brief Title
Safety Evaluation Study for Patients With Polycythemia Vera
Official Title
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
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Recruiting
Study Start Date
August 24, 2022 (Actual)
Primary Completion Date
August 1, 2023 (Anticipated)
Study Completion Date
March 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Perseus Proteomics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycythemia Vera
Keywords
Myeloproliferative neoplasms

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PPMX-T003
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
PPMX-T003
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study
Time Frame
45 weeks
Secondary Outcome Measure Information:
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Observed maximum plasma concentration [Cmax]
Time Frame
Day1,Day2,Day7
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Terminal elimination rate constant [λz]
Time Frame
Day1,Day2,Day7
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Area under the concentration-time curve from pre-dose [time 0] to the time of the last quantifiable concentration [AUC0-t]
Time Frame
Day1,Day2,Day7
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Area under the concentration-time curve from pre-dose [time 0] extrapolated to infinite time [AUC0-inf]
Time Frame
Day1,Day2,Day7
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Apparent terminal half-life [t½]
Time Frame
Day1,Day2,Day7
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Apparent systemic clearance [CL]
Time Frame
Day1,Day2,Day7
Title
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Description
Volume of distribution [Vd]
Time Frame
Day1,Day2,Day7
Title
Examining the expression rate of anti-drug antibodies (ADA)
Time Frame
Day1,Day21,up to 45weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
20 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Tadashi Matsuura
Phone
+81-3-5738-1705
Email
tmatsuura@ppmx.com
First Name & Middle Initial & Last Name or Official Title & Degree
Hiroko Akiyoshi
Phone
+81-3-5738-1705
Email
hakiyoshi@ppmx.com
Facility Information:
Facility Name
Kansai Medical University Hospital
City
Hirakata
State/Province
Osaka
ZIP/Postal Code
573-1191
Country
Japan
Individual Site Status
Recruiting
Facility Name
Shimane University Hospital
City
Izumo
State/Province
Shimane
ZIP/Postal Code
693-8501
Country
Japan
Individual Site Status
Recruiting
Facility Name
Osaka City University Hospital
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan
Individual Site Status
Recruiting

12. IPD Sharing Statement

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Safety Evaluation Study for Patients With Polycythemia Vera

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