臨床研究等提出・公開システム

Date of registration	Sept. 14, 2021
Last modified on	Mar. 17, 2025
Trial ID	jRCT2051210083

Scientific Title	Phase I, Open-Label, Multicentre, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of A Single Intravenous PPMX-T003 in Polycythemia Vera
Public Title	Safety evaluation study for patients with polycythemia vera

Contact for Scientific Queries	Name	Ito Tomoki
	Affiliation	KANSAI MEDICAL UNIVERSITY HOSPITAL
	Address	2-3-1, Shinmachi, Hirakata-shi, Osaka, 573-1191
	Telephone	+81-72-804-0101
	E-mail	itot@hirakata.kmu.ac.jp
Contact for Public Queries	Name	Matsuura Tadashi
	Affiliation	Perseus Proteomics Inc.
	Address	A.i. Nihombashi EAST 3F
	Telephone	+81-362648268
	E-mail	tmatsuura@ppmx.com

Recruitment status	Complete

Anticipated date of first enrollment		Sept. 15, 2021
Actual date of first enrollment		July. 27, 2022
Target sample size		6
Study Type		Interventional
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	uncontrolled control
	assignment	single assignment
	purpose	other
Key inclusion & exclusion criteria	Inclusion Criteria	1. Confirmed diagnosis of PV according to either the 2008 or 2016 WHO classification criteria 2. 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)
	Age Minimum	20age old over
	Age Maximum	75age old not
	Gender	Both
Health Condition(s) or Problem(s) Studied		Polycythemia Vera
Intervention(s)		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. 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. If phlebotomy is required for 4 to 12 weeks after administration, the next dose should be administered after phlebotomy.
Health Condition(s) Keyword		Myeloproliferative Neoplasms
Primary Outcome(s)		Number of patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the study (vital signs, Evaluation of laboratory test values, Standard 12-lead ECG, physical findings)
Secondary Outcome(s)		Pharmacokinetics: Measuring the concentration of PPMX-T003, calculate and assess each PK parameter Immunogenicity: Examining the expression rate and antibody titer of anti-drug antibodies (ADA) Pharmacodynamics: Assessment of the Red blood cell count, hemoglobin, hematocrit, reticulocytes, serum iron, ferritin, total iron binding capacity, transferrin saturation

Primary Sponsor	Perseus Proteomics Inc.

Name of Certified Review Board	Institutional Review Board of Kansai Medical University Hospital
Address	2-3-1, Shinmachi, Hirakata-shi, Osaka
Telephone	+81-72-804-2808
E-Mail	chiken@hirakata.kmu.ac.jp
Approval Status	Approval
Date of approval	Aug. 19, 2021

Name of Certified Review Board	Osaka Metropolitan University Hospital Institutional Review Board
Address	3-1-1 Umeda, Kita-ku, Osaka-shi, Osaka 123-4567, Japan, Osaka
Date of approval	Aug. 19, 2021

Name of Certified Review Board	Clinical Research Review Committee, Shimane University Hospital
Address	89-1 Enya-cho, Izumo-shi, Shimane 693-8501, Japan, Osaka
Date of approval	Aug. 19, 2021

Name of Certified Review Board	Tokai University Hospital Institutional Review Board
Address	143 Shimo-Kasuya, Isehara-shi, Kanagawa 259-1193, Japan, Osaka
Date of approval	Aug. 19, 2021

Name of Certified Review Board	Tokushukai Group Joint Institutional Review Board
Address	1-2-3 Kasumigaseki, Chiyoda-ku, Tokyo 102-8577, Japan, Osaka
Date of approval	Aug. 19, 2021

Name of Certified Review Board	Miyazaki University Hospital Institutional Review Board
Address	5200 Kihara, Kiyotake, Miyazaki 889-1692, Japan, Osaka
Date of approval	Aug. 19, 2021

Name of Certified Review Board	Joint Institutional Review Board of Iwate Prefecture Hospitals
Address	1-4-1 Ueda, Morioka, Iwate 020-0066, Japan, Osaka
Date of approval	Aug. 19, 2021

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
2	Mar. 17, 2025	(this page)	Changes
1	Sept. 14, 2021	Detail

List of change history