Safety Evaluation Study for Patients With Polycythemia Vera

NCT05074550 | Unknown Phase 1

• 1 other identifier: PPMX-T003-CT102
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

Polycythemia Vera

Keywords

Myeloproliferative neoplasms

Outcome Measures

Primary Outcomes (1)

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

  45 weeks

Secondary Outcomes (8)

• Evaluation of pharmacokinetics of PPMX-T003 after single dose

  Day1,Day2,Day7

• Evaluation of pharmacokinetics of PPMX-T003 after single dose

  Day1,Day2,Day7

• Evaluation of pharmacokinetics of PPMX-T003 after single dose

  Day1,Day2,Day7

• Evaluation of pharmacokinetics of PPMX-T003 after single dose

  Day1,Day2,Day7

• Evaluation of pharmacokinetics of PPMX-T003 after single dose

  Day1,Day2,Day7

Study Arms (1)

PPMX-T003

EXPERIMENTAL

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

Interventions

PPMX-T003 DRUG

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.

PPMX-T003

Eligibility Criteria

• Age: 20 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Patients administrated drugs for PV treatment such as hydroxyurea or ruxolitinib (aspirin is excluded)

Sponsors & Collaborators

• Perseus Proteomics Inc.: lead

Study Sites (3)

Kansai Medical University Hospital

Hirakata, Osaka, 573-1191, Japan

RECRUITING

Shimane University Hospital

Izumo, Shimane, 693-8501, Japan

RECRUITING

Osaka City University Hospital

Osaka, 545-8586, Japan

RECRUITING

Related Publications (1)

• Gorak EJ, Otterstatter M, Al Baghdadi T, Gillis N, Foran JM, Liu JJ, Bejar R, Gore SD, Kroft SH, Harrington A, Saber W, Starczynowski D, Rollison DE, Zhang L, Moscinski L, Wilson S, Thompson J, Borchert C, Sherman S, Hebert D, Walker ME, Padron E, DeZern AE, Sekeres MA. Discordant pathologic diagnoses of myelodysplastic neoplasms and their implications for registries and therapies. Blood Adv. 2023 Oct 24;7(20):6120-6129. doi: 10.1182/bloodadvances.2023010061.

  PMID: 37552083 DERIVED

MeSH Terms

Conditions

Polycythemia Vera; Myeloproliferative Disorders

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Tadashi Matsuura

CONTACT

+81-3-5738-1705; tmatsuura@ppmx.com

Hiroko Akiyoshi

CONTACT

+81-3-5738-1705; hakiyoshi@ppmx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2021
• First Posted: October 12, 2021
• Study Start: August 24, 2022
• Primary Completion: August 1, 2023
• Study Completion: March 31, 2024
• Last Updated: July 24, 2023

Record last verified: 2022-11

Locations

JP (3)