Polycythemia VeraPhase 1NCT05074550
Safety Evaluation Study for Patients With Polycythemia Vera
A Phase 1 study testing PPMX-T003 in people with Polycythemia Vera, run by Perseus Proteomics Inc. at 3 sites in Japan.
Trial summary
This is PhaseI Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of PPMX-T003 in Polycythemia Vera Read more about this study
- Who is it for?
- Ages 20 to 75 years
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- PPMX-T003
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesJapan
- Study dates
- 24 Aug 2022to 31 Mar 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Patients administrated drugs for PV treatment such as hydroxyurea or ruxolitinib (aspirin is excluded)
Trial locations and contacts
3 sites in 1 country.
Japan3 sites, 3 recruiting
- Recruiting
Kansai Medical University Hospital
Hirakata, Osaka, 573-1191
- Recruiting
Shimane University Hospital
Izumo, Shimane, 693-8501
- Recruiting
Osaka City University Hospital
Osaka, 545-8586
About this study
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
This is PhaseI Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of PPMX-T003 in Polycythemia Vera
- Conditions
- Polycythemia Vera
- Being tested
- PPMX-T003
- Keywords
- Myeloproliferative neoplasms
- Medical subject headings
- Polycythemia Vera, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PPMX-T003Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: PPMX-T003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study
Measured: 45 weeks
Secondary outcomes (8)
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Observed maximum plasma concentration [Cmax]
Measured: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Terminal elimination rate constant [λz]
Measured: 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]
Measured: 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]
Measured: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Apparent terminal half-life [t½]
Measured: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Apparent systemic clearance [CL]
Measured: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Volume of distribution [Vd]
Measured: Day1,Day2,Day7
Examining the expression rate of anti-drug antibodies (ADA)
Measured: Day1,Day21,up to 45weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2021
- Study start24 Aug 2022
- Primary completion1 Aug 2023 (estimated)
- Study completion31 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Polycythemia VeraStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Perseus Proteomics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05074550
- Sponsor's protocol ID
- PPMX-T003-CT102
- Status last verified
- Nov 2022
- First submitted
- 14 Sep 2021
- Last updated
- 24 Jul 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 37552083
Study at a glance
From the registry record, last updated by the sponsor on 24 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05074550
- Acronym / sponsor ID
- PPMX-T003-CT102
- Conditions
- Polycythemia Vera
- Keywords
- Myeloproliferative neoplasms
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Perseus Proteomics Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Aug 2022 (actual)
- Primary completion
- 1 Aug 2023 (estimated)
- Study completion
- 31 Mar 2024 (estimated)
- First posted
- 12 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is PhaseI Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of PPMX-T003 in Polycythemia Vera
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PPMX-T003 | Experimental | Drug: PPMX-T003This 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. |
Who may qualify
- Ages
- 20 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- Patients administrated drugs for PV treatment such as hydroxyurea or ruxolitinib (aspirin is excluded)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PPMX-T003As 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. | Drug | PPMX-T003 |
Primary outcome
Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study
Time frame: 45 weeks
8 secondary outcomes
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Evaluation of pharmacokinetics of PPMX-T003 after single dose
Time frame: Day1,Day2,Day7
Examining the expression rate of anti-drug antibodies (ADA)
Time frame: Day1,Day21,up to 45weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jul 2023.
- Derived from this study: PubMed 37552083Gorak 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PPMX-T003 in people with Polycythemia Vera, run by Perseus Proteomics Inc. at 3 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Tadashi Matsuura (+81-3-5738-1705, tmatsuura@ppmx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Experiencing Treatment-emergent Adverse Events (TEAEs) in the Study (time frame: 45 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05074550, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jul 2023.