Acute Myeloid LeukemiaPhase 1NCT05659732
A Study of PEP07 (Checkpoint Kinase 1 Inhibitor) in Patients With Advanced Cancer
A Phase 1 study testing PEP07 in people with Acute Myeloid Leukemia and Lymphoma, Mantle-Cell, run by PharmaEngine at 5 sites in 2 countries.
Trial summary
The goal of this clinical trial is To assess the safety and tolerability of PEP07 administered orally as a single dose and at escalating dose levels, and, to determine the dose-limiting toxicity (DLT) of study treatment in patients with Acute Myeloid Leukemia (AML) and Mantle Cell Lymphoma (MCL). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Lymphoma, Mantle-Cell
- What is being tested?
- PEP07
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 17 Jul 2023to 31 Dec 2026
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be ≥ 18 years of age.
- Must have histological or cytological confirmation advanced hematologic malignancy including:
- Relapsed or refractory AML (by the 5th edition of World Health Organization [WHO] classification of Hematolymphoid tumors)
- OR Relapsed or refractory MCL and have received at least two prior lines of treatment, including chemoimmunotherapy and BTKi and at least 1 measurable site of disease according to Revised Response Criteria for Malignant Lymphoma.
- Must have Eastern Cooperative Oncology Group (ECOG) Performance score of 0 to 2.
- Must have adequate renal function as demonstrated by a calculated creatinine clearance ≥ 50 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula.
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- Must have adequate liver function as demonstrated by:
- aspartate aminotransferase (AST) ≤ 2.5 × ULN
- alanine aminotransferase (ALT) ≤ 2.5 × ULN
- bilirubin ≤ 1.5 × ULN (unless considered due to leukemic organ involvement. Patients with Gilbert's Syndrome may have had a bilirubin > 1.5 × ULN per discussion between the PI or designee and sponsor)
- Left ventricular ejection fraction (LVEF) ≥ 50% measured by multiple-gated acquisition (MUGA) or echocardiogram.
- Previous AEs have been improved to baseline or Grade ≤ 1 NCI CTCAE v5.0.
- Female patients with reproductive potential must have a negative serum pregnancy test 7 days prior to the administration of PEP07.
- Patients will be required to have a Covid negative test either via reverse transcriptase polymerase chain reaction (RTPCR) or a rapid antigen test (RAT) test on Day -7/Day 1.
- Provision of signed and dated informed consent form.
Who cannot join
- Pregnant or breastfeeding females
- Females of childbearing potential and males whose partners are of childbearing potential who do not agree to the use of 2 forms of highly effective contraception during the treatment period and for 120 days after the last dose of PEP07.
- Patients who have received anti-cancer therapy including chemotherapy, radiotherapy, hormonal, or any investigational therapy within 14 days or 5 half-lives (whichever is shorter), or immunotherapy within 30 days prior to the first dose of PEP07. Patients who have received hydroxyurea or dexamethasone at any time prior to the start of study treatment is an exception to this criterion.
- Patients who have undergone allogeneic hematopoietic stem cell transplant (HSCT) within 60 days of PEP07 treatment.
- Patients who have received strong or moderate CYP3A4 inhibitors or inducers such as ketoconazole, erythromycin, netupitant, isavuconazole etc. within 5 half-lives or 7 days (whichever is the shortest) prior to the initiation of study treatment.
- Viral infection with HIV or viral hepatitis type B or C which require antiviral therapy and/or have positive serology test of hepatitis B surface antigen [HBsAg (+)] with HBV DNA ≥ 1000 IU/mL, or hepatitis C virus antibody [anti-HCV Ab (+)] with HCV RNA (+). If a patient is HBsAg (+) then HBV DNA needs to be tested. If a patient is anti-HCV Ab (+) then the patient needs to be followed for HCV RNA (-) to be enrolled.
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- Uncontrolled systemic infection /or requiring isolation.
- Patients with previous history of other malignant diseases within the last 5 years (other than adequately treated non-melanotic skin cancer, in-situ carcinoma of the uterine cervix or myelodysplastic syndromes).
- Patients with ongoing ≥ Grade 2 (CTCAE v5.0) toxicity (except alopecia and hot flashes) related to previous treatment.
- Patients with baseline QTc interval > 450 msec (i.e., CTCAE Grade ≥ 2) at screening (within 28 days prior to 1st dose of PEP07, mean of triplicate readings within approximately 5 minutes).
- Patients with cardiovascular disability status of New York Heart Association (NYHA) ≥ Class III, left ventricular ejection fraction < 45 % at baseline, history of cardiac ischemia within the past 6 months, or prior history of cardiac arrhythmia requiring treatment.
- Patients who have undergone any major surgery within 3 weeks prior to first study drug administration after enrollment.
- Patients with known active central nervous system (CNS) or leptomeningeal involvement.
- Patients who have had any of the following within 6 months prior to first administration of PEP07 after enrollment: myocardial infarction, severe/unstable angina pectoris, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, or seizure disorder.
- Have a history of hypersensitivity reactions or allergic reactions to PEP07 excipients and components.
- Patients with other medical or psychiatric condition that, in the opinion of the investigator, might interfere with the patient's participation in the trial or interfere with the interpretation of trial results.
- Others who are ineligible to participate in this clinical study as determined by the PI or designee.
Trial locations and contacts
5 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 2 countries
Taiwan4 sites, 4 recruiting
- Recruiting
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City
Hui-Hua Hsiao, MD
- Recruiting
China Medical University Hospital
Taichung
Su-Peng Yeh, MD
- Recruiting
Chang Gung Memorial Hospital- Linkou
Taipei
Hsiao-Wen Kao, MD
- Recruiting
National Taiwan University Hospital
Taipei
Hsin-An Hou, MD
Australia1 site, 1 recruiting
- Recruiting
Monash Medical Centre
Clayton, Victoria, 3168
Annette Opat, 03 9594 4044, annette.opat@monashhealth.org
Jake Shortt
About this study
Official title: A Phase 1b Study of PEP07 (Checkpoint Kinase 1 Inhibitor) in Patients With Advanced Cancer
The goal of this clinical trial is
- To assess the safety and tolerability of PEP07 administered orally as a single dose and at escalating dose levels, and, to determine the dose-limiting toxicity (DLT) of study treatment in patients with Acute Myeloid Leukemia (AML) and Mantle Cell Lymphoma (MCL).
- To determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of PEP07 monotherapy.
Participants will receive PEP07 administered orally once daily (QD) for 2 consecutive days and 5 days off, every week for 4 weeks until disease progression, intolerable toxicity, confirmed pregnancy, death, consent withdrawal, HSCT or other anti-cancer treatment is required, or the Sponsor ends the study, whichever occurs first.
Read the detailed description
This is a Phase 1b, open-label, multi-center study recruiting patients with R/R AML and MCL.
The study will utilize an Accelerated Titration Design in the lower dose levels followed by a traditional 3+3 dose escalation design at higher dose levels until RP2D is determined. The starting dose will be 40 mg.
All potential study candidates will provide informed consent and will undergo screening procedures before participating in the study. After a screening period of up to 28 days, qualified patients will be enrolled to receive their assigned dose regimen of PEP07 monotherapy.
- Conditions
- Acute Myeloid Leukemia, Lymphoma, Mantle-Cell
- Being tested
- PEP07
- Medical subject headings
- Leukemia, Myeloid, Acute, Lymphoma, Mantle-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PEP07Drug
PEP07 is a selective Chk1 inhibitor maleate drug which is supplied as a hard gelatin capsule. PEP07 will be provided as 20 mg and 150 mg strength capsules to be administrated orally. Patients allocated to different dose levels of PEP07 monotherapy will receive either 20 mg or 150 mg oral capsules for 2 consecutive days followed by 5 days treatment off (2-on/5-off) schedule every week, 4 weeks as a treatment cycle.
| Study group | Role | What they receive |
|---|---|---|
| PEP07 Monotherapy (Arm A) | Experimental | The dose finding of PEP07 monotherapy consists of an accelerated dose escalation followed by a standard "3+3" dose escalation. Accelerated titration will continue until 1 patient experiences a DLT, or ≥ grade 2 AE related to PEP07 (except for hematologic toxicities) at any dose level, after which, additional 2 patients will be enrolled in the same cohort, and the study will be switched to a 3+3 scheme.
Expansion cohorts with 12 patients will be opened for Arm A at the RP2D once efficacy signal is observed at this level. Receives: Drug: PEP07 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the safety and tolerability of PEP07 administered orally as a single dose and at escalating dose levels, and, to determine the dose-limiting toxicity (DLT) of study treatment in patients with AML and MCL.
to find out the DLT
Measured: 4 weeks after first dosing
To determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of PEP07 monotherapy.
to find out the MTD
Measured: 4 weeks after first dosing
Secondary outcomes (6)
To assess the AUC from time zero to infinity (AUC0-inf) of single and multiple oral PEP07 as a monotherapy.
to know the PK profile
Measured: 6 months
To assess the Time to maximum (peak) plasma concentration (Tmax) of single and multiple oral PEP07 as a monotherapy.
to know the PK profile
Measured: 6 months
To assess the apparent half-life (t1/2) of single and multiple oral PEP07 as a monotherapy.
to know the PK profile
Measured: 6 months
To assess the Maximum observed concentration (Cmax) of single and multiple oral PEP07 as a monotherapy.
to know the PK profile
Measured: 6 months
To assess preliminary evidence of anti-tumor activity of PEP07 by progression free survival (PFS).
to know the efficacy
Measured: after first dosing to disease progression
To assess preliminary evidence of anti-tumor activity of PEP07 by progression free survival (PFS).
to know the efficacy
Measured: after first dosing to disease progress
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2022
- Study start17 Jul 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PharmaEngine
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hsin-An Hou, M.D., Principal investigator, Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05659732
- Sponsor's protocol ID
- PEP07-101
- Status last verified
- Jul 2025
- First submitted
- 30 Nov 2022
- Last updated
- 31 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Study of PEP07 (Checkpoint Kinase 1 Inhibitor) in Patients With Advanced Cancer
- ClinicalTrials.gov ID
- NCT05659732
- Acronym / sponsor ID
- PEP07-101
- Conditions
- Acute Myeloid Leukemia, Lymphoma, Mantle-Cell
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- PharmaEngineIndustry (pharma, biotech, devices)
- Study start
- 17 Jul 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 21 Dec 2022 (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.
The goal of this clinical trial is
- To assess the safety and tolerability of PEP07 administered orally as a single dose and at escalating dose levels, and, to determine the dose-limiting toxicity (DLT) of study treatment in patients with Acute Myeloid Leukemia (AML) and Mantle Cell Lymphoma (MCL).
- To determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of PEP07 monotherapy.
Participants will receive PEP07 administered orally once daily (QD) for 2 consecutive days and 5 days off, every week for 4 weeks until disease progression, intolerable toxicity, confirmed pregnancy, death, consent withdrawal, HSCT or other anti-cancer treatment is required, or the Sponsor ends the study, whichever occurs first.
Detailed description
This is a Phase 1b, open-label, multi-center study recruiting patients with R/R AML and MCL.
The study will utilize an Accelerated Titration Design in the lower dose levels followed by a traditional 3+3 dose escalation design at higher dose levels until RP2D is determined. The starting dose will be 40 mg.
All potential study candidates will provide informed consent and will undergo screening procedures before participating in the study. After a screening period of up to 28 days, qualified patients will be enrolled to receive their assigned dose regimen of PEP07 monotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PEP07 Monotherapy (Arm A) | Experimental | Drug: PEP07The dose finding of PEP07 monotherapy consists of an accelerated dose escalation followed by a standard "3+3" dose escalation. Accelerated titration will continue until 1 patient experiences a DLT, or ≥ grade 2 AE related to PEP07 (except for hematologic toxicities) at any dose level, after which, additional 2 patients will be enrolled in the same cohort, and the study will be switched to a 3+3 scheme. Expansion cohorts with 12 patients will be opened for Arm A at the RP2D once efficacy signal is observed at this level. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Must be ≥ 18 years of age.
- Must have histological or cytological confirmation advanced hematologic malignancy including:
- Must have Eastern Cooperative Oncology Group (ECOG) Performance score of 0 to 2.
- Must have adequate renal function as demonstrated by a calculated creatinine clearance ≥ 50 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula.
- Must have adequate liver function as demonstrated by:
Key exclusion criteria (17 listed)
- Pregnant or breastfeeding females
- Females of childbearing potential and males whose partners are of childbearing potential who do not agree to the use of 2 forms of highly effective contraception during the treatment period and for 120 days after the last dose of PEP07.
- Patients who have received anti-cancer therapy including chemotherapy, radiotherapy, hormonal, or any investigational therapy within 14 days or 5 half-lives (whichever is shorter), or immunotherapy within 30 days prior to the first dose of PEP07. Patients who have received hydroxyurea or dexamethasone at any time prior to the start of study treatment is an exception to this criterion.
- Patients who have undergone allogeneic hematopoietic stem cell transplant (HSCT) within 60 days of PEP07 treatment.
- Patients who have received strong or moderate CYP3A4 inhibitors or inducers such as ketoconazole, erythromycin, netupitant, isavuconazole etc. within 5 half-lives or 7 days (whichever is the shortest) prior to the initiation of study treatment.
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 |
|---|---|---|
| PEP07PEP07 is a selective Chk1 inhibitor maleate drug which is supplied as a hard gelatin capsule. PEP07 will be provided as 20 mg and 150 mg strength capsules to be administrated orally. Patients allocated to different dose levels of PEP07 monotherapy will receive either 20 mg or 150 mg oral capsules for 2 consecutive days followed by 5 days treatment off (2-on/5-off) schedule every week, 4 weeks as a treatment cycle. | Drug | PEP07 Monotherapy (Arm A) |
Primary outcome
To assess the safety and tolerability of PEP07 administered orally as a single dose and at escalating dose levels, and, to determine the dose-limiting toxicity (DLT) of study treatment in patients with AML and MCL.
Time frame: 4 weeks after first dosing
to find out the DLT
To determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of PEP07 monotherapy.
Time frame: 4 weeks after first dosing
to find out the MTD
6 secondary outcomes
To assess the AUC from time zero to infinity (AUC0-inf) of single and multiple oral PEP07 as a monotherapy.
Time frame: 6 months
To assess the Time to maximum (peak) plasma concentration (Tmax) of single and multiple oral PEP07 as a monotherapy.
Time frame: 6 months
To assess the apparent half-life (t1/2) of single and multiple oral PEP07 as a monotherapy.
Time frame: 6 months
To assess the Maximum observed concentration (Cmax) of single and multiple oral PEP07 as a monotherapy.
Time frame: 6 months
To assess preliminary evidence of anti-tumor activity of PEP07 by progression free survival (PFS).
Time frame: after first dosing to disease progression
To assess preliminary evidence of anti-tumor activity of PEP07 by progression free survival (PFS).
Time frame: after first dosing to disease progress
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 31 Jul 2025.
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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.
Hsin-An Hou, M.D. principal investigator
Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PEP07 in people with Acute Myeloid Leukemia and Lymphoma, Mantle-Cell, run by PharmaEngine at 5 sites in 2 countries.
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 15 inclusion and 17 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 31 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Taiwan, Australia. 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?
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 Brian Shen (+886 2 2515-8228, brian.shen@pharmaengine.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the safety and tolerability of PEP07 administered orally as a single dose and at escalating dose levels, and, to determine the dose-limiting toxicity (DLT) of study treatment in patients with AML and MCL. (time frame: 4 weeks after first dosing). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Jul 2025.