Aggressive NK Cell LeukemiaPhase 1/2NCT05863234
Safety Evaluation Study for Patients With Aggressive NK-cell Leukemia
A Phase 1/2 study testing PPMX-T003 in people with Aggressive NK Cell Leukemia, run by Hiroshima University Hospital at 1 site in Japan.
Trial summary
This is Phase I/II Dose-Escalation Study to evaluate the tolerability, safety, efficacy and pharmacokinetics of PPMX-T003 in aggressive NK-cell leukemia. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Aggressive NK Cell Leukemia
- What is being tested?
- PPMX-T003
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteJapan
- Study dates
- 21 Sep 2023to 31 Mar 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients diagnosed with ANKL (regardless of whether the disease is first or recurrent) based on diagnostic criteria developed with reference to the World Health Organization (WHO) 4th edition (2017) criteria.
Who cannot join
- Patients eligible to receive chemotherapy as treatment for ANKL
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Japan1 site, 1 recruiting
- Recruiting
Hiroshima University Hospital
Hiroshima, Hiroshima, 734-8551
Kiyoshi Ando, +81 82-257-5555, andok@keyaki.cc.u-tokai.ac.jp
About this study
Official title: Multicenter, Open-label, Dose-escalation Phase I/II Study to Evaluate the Tolerability, Safety, Efficacy and Pharmacokinetics of Repeated Continuous Intravenous PPMX-T003 in Patients With Aggressive NK Cell Leukaemia (ANKL) (Physician-initiated Clinical Trial)
This is Phase I/II Dose-Escalation Study to evaluate the tolerability, safety, efficacy and pharmacokinetics of PPMX-T003 in aggressive NK-cell leukemia.
- Conditions
- Aggressive NK Cell Leukemia
- Being tested
- PPMX-T003
- Keywords
- neoplasms
- Medical subject headings
- Leukemia, Large Granular Lymphocytic, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PPMX-T003Drug
The therapeutic agent is administered continuously intravenously
| Study group | Role | What they receive |
|---|---|---|
| Treatment with PPMX-T003 | Experimental | Not described Receives: Drug: PPMX-T003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of incidence of AEs and DLTs by PPMX-T003 repeated continuous intravenous administration (the first course)
In order to evaluate tolerability and safety of PPMX-T003 repeated continuous intravenous administration in the first course, incidence of AEs and DLTs by the treatment are measured.
Measured: 7days after the administration
Secondary outcomes (7)
Number of incidence of AEs and DLTs by PPMX-T003 repeated continuous intravenous administration (the second course or later)
In order to evaluate tolerability and safety of PPMX-T003 repeated continuous intravenous administration in the second course or later, incidence of AEs and DLTs by the treatment are measured.
Measured: From the second course to the final course (Max. 35days after the first administration)
Efficacy of PPMX-T003 repeated continuous intravenous administration (1) Liver volume evaluated by CT scan
As a part of efficacy evaluation of the treatment, percentage change in the product of the maximum long and short diameters of the liver measured by CT (assessed by the primary physician)
Measured: 35 days
Efficacy of PPMX-T003 repeated continuous intravenous administration (2) improvement in liver function as assessed by the Model for End-stage Liver Disease (MELD and MELD-Na)
Measured: 35 days
Efficacy of PPMX-T003 repeated continuous intravenous administration (3) Survival duration at 6 months
Measured: 6 months
Efficacy of PPMX-T003 repeated continuous intravenous administration (4) Percentage of subjects who were able to transition to chemotherapy
Measured: 35days after the treatment
Serum drug concentration during repeated continuous intravenous PPMX-T003 administration
Measured: 35 days
Anti-drug antibody production (Immunogenicity)
Measured: 35 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2023
- Study start21 Sep 2023
- Primary completion31 Mar 2026 (estimated)
- Study completion31 Mar 2026 (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 Aggressive NK Cell LeukemiaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hiroshima University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Kyoto University Hospital, Hokkaido University Hospital, Okayama University, Tokai University, Kyushu University, Tohoku University, Nagoya University, Komagome Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05863234
- Sponsor's protocol ID
- PPMX-T003-CT103
- Status last verified
- Apr 2025
- First submitted
- 9 May 2023
- Last updated
- 30 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Fukuhara N, Onizuka M, Kanda J, Asada N, Kato K, Ando K. Protocol for a multicentre, open-label, dose-escalation phase I/II study evaluating the tolerability, safety, efficacy and pharmacokinetics of repeated continuous intravenous PPMX-T003 in patients with aggressive natural killer cell leukaemia. BMJ Open. 2025 Jun 30;15(6):e098532. doi: 10.1136/bmjopen-2024-098532.
PubMed 40588385
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Open-label, Dose-escalation Phase I/II Study to Evaluate the Tolerability, Safety, Efficacy and Pharmacokinetics of Repeated Continuous Intravenous PPMX-T003 in Patients With Aggressive NK Cell Leukaemia (ANKL) (Physician-initiated Clinical Trial)
- ClinicalTrials.gov ID
- NCT05863234
- Acronym / sponsor ID
- PPMX-T003-CT103
- Conditions
- Aggressive NK Cell Leukemia
- Keywords
- neoplasms
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- Hiroshima University HospitalUniversity, hospital or other
- Collaborators
- Kyoto University Hospital, Hokkaido University Hospital, Okayama University, Tokai University, Kyushu University, Tohoku University, Nagoya University, Komagome Hospital
- Study start
- 21 Sep 2023 (actual)
- Primary completion
- 31 Mar 2026 (estimated)
- Study completion
- 31 Mar 2026 (estimated)
- First posted
- 17 May 2023 (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 Phase I/II Dose-Escalation Study to evaluate the tolerability, safety, efficacy and pharmacokinetics of PPMX-T003 in aggressive NK-cell leukemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with PPMX-T003 | Experimental | Drug: PPMX-T003 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Patients diagnosed with ANKL (regardless of whether the disease is first or recurrent) based on diagnostic criteria developed with reference to the World Health Organization (WHO) 4th edition (2017) criteria.
Key exclusion criteria (1 listed)
- Patients eligible to receive chemotherapy as treatment for ANKL
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-T003The therapeutic agent is administered continuously intravenously | Drug | Treatment with PPMX-T003 |
Primary outcome
Number of incidence of AEs and DLTs by PPMX-T003 repeated continuous intravenous administration (the first course)
Time frame: 7days after the administration
In order to evaluate tolerability and safety of PPMX-T003 repeated continuous intravenous administration in the first course, incidence of AEs and DLTs by the treatment are measured.
7 secondary outcomes
Number of incidence of AEs and DLTs by PPMX-T003 repeated continuous intravenous administration (the second course or later)
Time frame: From the second course to the final course (Max. 35days after the first administration)
Efficacy of PPMX-T003 repeated continuous intravenous administration (1) Liver volume evaluated by CT scan
Time frame: 35 days
Efficacy of PPMX-T003 repeated continuous intravenous administration (2) improvement in liver function as assessed by the Model for End-stage Liver Disease (MELD and MELD-Na)
Time frame: 35 days
Efficacy of PPMX-T003 repeated continuous intravenous administration (3) Survival duration at 6 months
Time frame: 6 months
Efficacy of PPMX-T003 repeated continuous intravenous administration (4) Percentage of subjects who were able to transition to chemotherapy
Time frame: 35days after the treatment
Serum drug concentration during repeated continuous intravenous PPMX-T003 administration
Time frame: 35 days
Anti-drug antibody production (Immunogenicity)
Time frame: 35 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2025.
- Derived from this study: PubMed 40588385Fukuhara N, Onizuka M, Kanda J, Asada N, Kato K, Ando K. Protocol for a multicentre, open-label, dose-escalation phase I/II study evaluating the tolerability, safety, efficacy and pharmacokinetics of repeated continuous intravenous PPMX-T003 in patients with aggressive natural killer cell leukaemia. BMJ Open. 2025 Jun 30;15(6):e098532. doi: 10.1136/bmjopen-2024-098532.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PPMX-T003 in people with Aggressive NK Cell Leukemia, run by Hiroshima University Hospital at 1 site 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 1 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: looking for participants now, as of the sponsor's last update on 30 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kiyoshi Ando (+81 82-257-5555, andok@keyaki.cc.u-tokai.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of incidence of AEs and DLTs by PPMX-T003 repeated continuous intravenous administration (the first course) (time frame: 7days after the administration). 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 NCT05863234, 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 30 Apr 2025.