Safety Evaluation Study for Patients With Polycythemia Vera

Sponsor

Perseus Proteomics Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05074550

Collaborator

(none)

Enrollment

Locations

Arm

Anticipated Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Polycythemia Vera	Drug: PPMX-T003	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

6 participants

Allocation:

N/A

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Jul 1, 2022

Anticipated Primary Completion Date :

Oct 1, 2023

Anticipated Study Completion Date :

Oct 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
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.	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.

Arm

Intervention/Treatment

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.

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.

Outcome Measures

Primary Outcome Measures

Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study [45 weeks]

Secondary Outcome Measures

Evaluation of pharmacokinetics of PPMX-T003 after single dose [Day1,Day2,Day7]
Observed maximum plasma concentration [Cmax]
Evaluation of pharmacokinetics of PPMX-T003 after single dose [Day1,Day2,Day7]
Terminal elimination rate constant [λz]
Evaluation of pharmacokinetics of PPMX-T003 after single dose [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 [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 [Day1,Day2,Day7]
Apparent terminal half-life [t½]
Evaluation of pharmacokinetics of PPMX-T003 after single dose [Day1,Day2,Day7]
Apparent systemic clearance [CL]
Evaluation of pharmacokinetics of PPMX-T003 after single dose [Day1,Day2,Day7]
Volume of distribution [Vd]
Examining the expression rate of anti-drug antibodies (ADA) [Day1,Day21,up to 45weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

20 Years to 75 Years

Sexes Eligible for Study:

All

Accepts 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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Kansai Medical University Hospital	Hirakata	Osaka	Japan	573-1191
2	Shimane University Hospital	Izumo	Shimane	Japan	693-8501
3	Osaka City University Hospital	Osaka		Japan	545-8586

Sponsors and Collaborators

Perseus Proteomics Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Perseus Proteomics Inc.

ClinicalTrials.gov Identifier:

NCT05074550

Other Study ID Numbers:

PPMX-T003-CT102

First Posted:

Oct 12, 2021

Last Update Posted:

Jul 1, 2022

Last Verified:

Oct 1, 2021

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Perseus Proteomics Inc.

Myeloproliferative neoplasms

Additional relevant MeSH terms:

Polycythemia Vera

Polycythemia

Study Results

No Results Posted as of Jul 1, 2022