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This is PhaseI Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of PPMX-T003 in Polycythemia Vera
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study | 45 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Observed maximum plasma concentration [Cmax] | Day1,Day2,Day7 |
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Terminal elimination rate constant [λz] |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Tadashi Matsuura | Contact | +81-3-5738-1705 | tmatsuura@ppmx.com | |
| Hiroko Akiyoshi | Contact | +81-3-5738-1705 | hakiyoshi@ppmx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kansai Medical University Hospital | Recruiting | Hirakata | Osaka | 573-1191 | Japan | |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37552083 | Derived | 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. |
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| ID | Term |
|---|---|
| D011087 | Polycythemia Vera |
| D009196 | Myeloproliferative Disorders |
| ID | Term |
|---|---|
| D019046 | Bone Marrow Neoplasms |
| D019337 | Hematologic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
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| Day1,Day2,Day7 |
| 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] | Day1,Day2,Day7 |
| 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] | Day1,Day2,Day7 |
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Apparent terminal half-life [t½] | Day1,Day2,Day7 |
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Apparent systemic clearance [CL] | Day1,Day2,Day7 |
| Evaluation of pharmacokinetics of PPMX-T003 after single dose | Volume of distribution [Vd] | Day1,Day2,Day7 |
| Examining the expression rate of anti-drug antibodies (ADA) | Day1,Day21,up to 45weeks |
| Shimane University Hospital |
| Recruiting |
| Izumo |
| Shimane |
| 693-8501 |
| Japan |
| Osaka City University Hospital | Recruiting | Osaka | 545-8586 | Japan |
| D001855 |
| Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |