AML (Acute Myelogenous LeukemiaPhase 1NCT07014449
Clinical Trial of WBC100 Capsule in Relapsed/Refractory Acute Myeloid Leukemia
A Phase 1 study testing WBC100 QD in people with AML (Acute Myelogenous Leukemia and Relapsed Acute Myelogenous Leukemia, run by Hangzhou Weben Pharma Co., Ltd at 1 site in China.
Trial summary
The goal of this clinical trial is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of WBC100 capsules in patients with relapsed or refractory acute myeloid leukemia (R/R AML). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML (Acute Myelogenous Leukemia, Relapsed Acute Myelogenous Leukemia, Refractory Acute Myeloid Leukemia
- What is being tested?
- WBC100 QD
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteChina
- Study dates
- 3 Nov 2024to 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
- 1. Signed informed consent and compliance with study procedures;
- 2. Male or female participants aged ≥18 years at the time of consent;
- 3. Diagnosis of relapsed or refractory acute myeloid leukemia (R/R AML) according to the 2016 World Health Organization (WHO) classification;
- 4. ECOG PS 0-2;
- 5. Life expectancy ≥3 months;
- 6. Adequate bone marrow reserve and organ function as defined below:
Show all 12Show fewer
- Bone marrow reserve: Peripheral WBC < 25 × 10⁹/L (leukocyte-reducing agents are allowed, with a washout period of at least 5 half-lives prior to study drug administration);
- Coagulation: International normalized ratio (INR) ≤ 2;
- Hepatic function: Total bilirubin (TBIL) ≤ 1.5 × ULN; ALT and AST ≤ 2.5 × ULN. In cases of hepatic involvement: ALT or AST ≤ 5 × ULN, and TBIL ≤ 3 × ULN;
- Renal function: Creatinine clearance ≥60 mL/min (Cockcroft-Gault), or serum creatinine ≤1.5 × ULN;
- Cardiac function: Left ventricular ejection fraction (LVEF) ≥50%; QTcF ≤450 ms for males, ≤470 ms for females.
- 7. Female participants of childbearing potential and fertile male participants with partners of childbearing potential must use medically approved contraception during treatment and for 6 months after the final dose.
Who cannot join
- 1. Known hypersensitivity to WBC100 capsules or any of their excipients;
- 2. Diagnosis of acute promyelocytic leukemia (APL);
- 3. Diagnosis of mixed phenotype acute leukemia, chronic myeloid leukemia in blast crisis, or AML transformed from myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN);
- 4. Subjects with relapse after allogeneic HSCT, grade ≥ 2 acute GVHD, extensive chronic GVHD requiring immunosuppressive therapy, or autologous HSCT within the past 90 days;
- 5. Subjects who have undergone major surgery, have active ulcers, or have unhealed wounds within 28 days prior to the first dose;
- 6. Received other investigational drugs or treatments within 28 days prior to the first administration, or are still within the safety follow-up period of another clinical trial;
Show all 26Show fewer
- 7. Subjects with a history of severe cardiovascular or cerebrovascular conditions, including but not limited to:
- Significant arrhythmias or conduction disorders (e.g., ventricular arrhythmias, Grade II-III AV block);
- Thromboembolic events requiring anticoagulation or presence of vena cava filter;
- NYHA Class III-IV heart failure;
- Poorly controlled hypertension (SBP ≥140 mmHg or DBP ≥90 mmHg despite treatment).
- 8. Evidence of severe or uncontrolled systemic diseases, such as refractory effusions, poorly controlled diabetes, or significant disorders of the psychiatric, neurological, cardiovascular, respiratory, endocrine, gastrointestinal, hepatic, or renal systems;
- 9. History or presence of immunodeficiency, autoimmune disease requiring systemic immunosuppressants, or organ transplantation;
- 10. Congestive heart failure, aortic dissection, stroke (excluding lacunar infarct), unstable angina, myocardial infarction, bypass surgery, or pulmonary embolism within 180 days prior to first dosing;
- 11. Known risk factors for QT prolongation, including congenital long QT syndrome or drug-induced arrhythmia history;
- 12. Positive for syphilis antibodies, HIV, active HBV infection (HBsAg+ or HBcAb+ with HBV DNA ≥1000 IU/mL), or active HCV infection (HCV Ab+ with detectable HCV RNA);
- 13. Active infection requiring systemic treatment, including uncontrolled bacterial, viral, or fungal infections;
- 14. Gastrointestinal conditions preventing oral drug intake or absorption, such as severe vomiting, chronic diarrhea, intestinal stoma, malabsorption, or inability to swallow;
- 15. Use of strong CYP450 inhibitors/inducers that cannot be stopped ≥7 days before dosing;
- 16. Receipt of monoclonal antibodies, ADCs, radiotherapy within 28 days (14 days for localized radiotherapy), cytotoxic chemotherapy, targeted small molecules within 14 days or 5 half-lives, or CAR-T therapy within 100 days;
- 17. Receipt of any live or attenuated vaccines (e.g., influenza, varicella) within 28 days;
- 18. History of other malignancies within 2 years, except adequately treated basal cell carcinoma, carcinoma in situ of cervix or breast, or squamous cell carcinoma of the skin;
- 19. History of psychiatric or neurological disorders that may interfere with protocol compliance;
- 20. Inability to tolerate venous blood draws;
- 21. Pregnant or breastfeeding women, or women with positive serum hCG during screening;
- 22. Any condition deemed by the investigator to make the subject unsuitable for study participation.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital, School of Medicine, Zhejiang University
Hangzhou, Zhejiang, 310003
Jie Jin, M.D., +86 571-87236896, jiej0503@163.com
About this study
Official title: An Open-Label, Phase I Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetic, and Preliminary Efficacy of WBC100 Capsules in Patients With Relapsed or Refractory Acute Myeloid Leukemia
The goal of this clinical trial is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of WBC100 capsules in patients with relapsed or refractory acute myeloid leukemia (R/R AML). The main questions it aims to answer are:
- What is the safety and tolerability profile of WBC100 in R/R AML patients?
- Can WBC100 effectively induce remission in R/R AML patients?
Read more
Participants will:
- Take WBC100 capsules orally once daily in 28-day treatment cycles;
- Undergo regular safety assessments, including adverse event monitoring and laboratory tests;
- Provide blood samples for pharmacokinetic (PK) analysis;
- Have their remission status and efficacy evaluated according to the ELN2022 criteria.
- Conditions
- AML (Acute Myelogenous Leukemia, Relapsed Acute Myelogenous Leukemia, Refractory Acute Myeloid Leukemia, Hematologic Malignancy, C-Myc, Adult Acute Myeloid Leukemia
- Being tested
- WBC100 QD
- Keywords
- c-Myc, Molecular glue, Acute Myelogenous Leukemia, Relapsed and Refractory Acute Myelogenous Leukemia
- Medical subject headings
- Leukemia, Myeloid, Acute, Hematologic Neoplasms, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WBC100 QDDrug
WBC100 will be administered orally as capsules once daily in 28-day cycles.
| Study group | Role | What they receive |
|---|---|---|
| WBC100 Dose Escalation Cohort | Experimental | Participants will take WBC100 capsules orally once daily in 28-day cycles. This arm includes a dose-escalation phase using an accelerated titration combined with the traditional '3+3' design. After a single-patient observation at 0.5 mg, three patients will be enrolled per cohort at each subsequent dose level (1.0, 1.5, 2.0, 2.5, and 3.0 mg). Treatment will continue until disease progression, unacceptable toxicity, or withdrawal. Receives: Drug: WBC100 QD |
| WBC100 2.5 mg QD Dose Expansion Cohort | Experimental | Participants will receive 2.5 mg of the investigational drug orally once daily (QD) during the dose expansion phase. Receives: Drug: WBC100 QD |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of dose-limiting toxicities (DLTs)
Evaluated according to NCI-CTCAE version 5.0.
Measured: During the first treatment cycle (28 days)
Incidence and severity of treatment-emergent adverse events (TEAEs)
Includes lab abnormalities, physical exam findings, vital signs, and ECG changes.
Measured: From first dose to 28 days after the last dose
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)
Based on observed DLTs.
Measured: 28 days
Secondary outcomes (20)
Cmax
Peak plasma concentration after one dose, measured using a validated LC-MS/MS method
Measured: 28 days
Tmax
Time to peak plasma concentration after one dose
Measured: 28 days
AUC0-t
Area under the plasma concentration versus time curve after one dose and multiple dose; time range from 0 to last point when plasma concentration is detectable
Measured: 28 days
AUC0-inf
Area under the plasma concentration versus time curve; time range from 0 to infinity
Measured: 28 days
T1/2
Half-life period
Measured: 28 days
λz
Elimination rate constant
Measured: 28 days
CL/F
Apparent clearance
Measured: 28 days
Vz/F
Apparent volume of distribution
Measured: 28 days
Cmax, ss
Steady peak plasma concentration after multiple dose
Measured: 28 days
Cmin, ss
Steady minimal plasma concentration after multiple dose
Measured: 28 days
Cavg
Steady average plasma concentration after multiple dose
Measured: 28 days
Tmax, ss
Time to steady peak plasma concentration after multiple dose
Measured: 28 days
CLss/F
Steady apparent clearance
Measured: 28 days
Vss/F
Steady apparent volume of distribution
Measured: 28 days
ARCmax
Peak concentration cumulative coefficient
Measured: 28 days
ARAUC
AUC cumulative coefficient
Measured: 28 days
DF
Degree of fluctuation (DF) will be calculated as (Cmax,ss - Cmin,ss) / Cavg,ss × 100%, where Cmax,ss is the maximum plasma concentration at steady state, Cmin,ss is the minimum plasma concentration at steady state, and Cavg,ss is the average plasma concentration over the dosing interval at steady state.
Measured: 28 days
Duration of response (DOR)
The period from the first evaluation of complete response (CR) or partial response (PR) to the first evaluation of progressive disease (PD) or death of any cause
Measured: 2 years
Event-free survival period (EFS)
EFS, defined as the time from the start of the subject's enrollment to the occurrence of any event (treatment failure/relapse after CR, CRh or CRi/permanent termination of treatment for any reason/death from any cause), whichever occurs first.
Measured: 2 years
Overall survival (OS)
From date of treatment start until the date of death due to any cause.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2025
- Study start3 Nov 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myelogenous 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hangzhou Weben Pharma Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07014449
- Sponsor's protocol ID
- WB002
- Status last verified
- Oct 2026
- First submitted
- 22 May 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Phase I Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetic, and Preliminary Efficacy of WBC100 Capsules in Patients With Relapsed or Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07014449
- Acronym / sponsor ID
- WB002
- Conditions
- AML (Acute Myelogenous Leukemia, Relapsed Acute Myelogenous Leukemia, Refractory Acute Myeloid Leukemia, Hematologic Malignancy, C-Myc, Adult Acute Myeloid Leukemia
- Keywords
- c-Myc, Molecular glue, Acute Myelogenous Leukemia, Relapsed and Refractory Acute Myelogenous Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Hangzhou Weben Pharma Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 3 Nov 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 11 Jun 2025 (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 evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of WBC100 capsules in patients with relapsed or refractory acute myeloid leukemia (R/R AML). The main questions it aims to answer are:
- What is the safety and tolerability profile of WBC100 in R/R AML patients?
- Can WBC100 effectively induce remission in R/R AML patients?
Participants will:
- Take WBC100 capsules orally once daily in 28-day treatment cycles;
- Undergo regular safety assessments, including adverse event monitoring and laboratory tests;
- Provide blood samples for pharmacokinetic (PK) analysis;
- Have their remission status and efficacy evaluated according to the ELN2022 criteria.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| WBC100 Dose Escalation Cohort | Experimental | Drug: WBC100 QDParticipants will take WBC100 capsules orally once daily in 28-day cycles. This arm includes a dose-escalation phase using an accelerated titration combined with the traditional '3+3' design. After a single-patient observation at 0.5 mg, three patients will be enrolled per cohort at each subsequent dose level (1.0, 1.5, 2.0, 2.5, and 3.0 mg). Treatment will continue until disease progression, unacceptable toxicity, or withdrawal. |
| WBC100 2.5 mg QD Dose Expansion Cohort | Experimental | Drug: WBC100 QDParticipants will receive 2.5 mg of the investigational drug orally once daily (QD) during the dose expansion phase. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 1. Signed informed consent and compliance with study procedures;
- 2. Male or female participants aged ≥18 years at the time of consent;
- 3. Diagnosis of relapsed or refractory acute myeloid leukemia (R/R AML) according to the 2016 World Health Organization (WHO) classification;
- 4. ECOG PS 0-2;
- 5. Life expectancy ≥3 months;
Key exclusion criteria (26 listed)
- 1. Known hypersensitivity to WBC100 capsules or any of their excipients;
- 2. Diagnosis of acute promyelocytic leukemia (APL);
- 3. Diagnosis of mixed phenotype acute leukemia, chronic myeloid leukemia in blast crisis, or AML transformed from myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN);
- 4. Subjects with relapse after allogeneic HSCT, grade ≥ 2 acute GVHD, extensive chronic GVHD requiring immunosuppressive therapy, or autologous HSCT within the past 90 days;
- 5. Subjects who have undergone major surgery, have active ulcers, or have unhealed wounds within 28 days prior to the first dose;
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 |
|---|---|---|
| WBC100 QDWBC100 will be administered orally as capsules once daily in 28-day cycles. | Drug | WBC100 2.5 mg QD Dose Expansion Cohort, WBC100 Dose Escalation Cohort |
Primary outcome
Incidence and severity of dose-limiting toxicities (DLTs)
Time frame: During the first treatment cycle (28 days)
Evaluated according to NCI-CTCAE version 5.0.
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time frame: From first dose to 28 days after the last dose
Includes lab abnormalities, physical exam findings, vital signs, and ECG changes.
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)
Time frame: 28 days
Based on observed DLTs.
20 secondary outcomes
Cmax
Time frame: 28 days
Tmax
Time frame: 28 days
AUC0-t
Time frame: 28 days
AUC0-inf
Time frame: 28 days
T1/2
Time frame: 28 days
λz
Time frame: 28 days
CL/F
Time frame: 28 days
Vz/F
Time frame: 28 days
Cmax, ss
Time frame: 28 days
Cmin, ss
Time frame: 28 days
Cavg
Time frame: 28 days
Tmax, ss
Time frame: 28 days
CLss/F
Time frame: 28 days
Vss/F
Time frame: 28 days
ARCmax
Time frame: 28 days
ARAUC
Time frame: 28 days
DF
Time frame: 28 days
Duration of response (DOR)
Time frame: 2 years
Event-free survival period (EFS)
Time frame: 2 years
Overall survival (OS)
Time frame: 2 years
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 16 Oct 2026.
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 study testing WBC100 QD in people with AML (Acute Myelogenous Leukemia and Relapsed Acute Myelogenous Leukemia, run by Hangzhou Weben Pharma Co., Ltd at 1 site in China.
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 12 inclusion and 26 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 16 Oct 2026. 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 China. 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 Wu Shuigao, M.S (+86-18761861260, sgwu@webenpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of dose-limiting toxicities (DLTs) (time frame: During the first treatment cycle (28 days)). The study lists 3 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 16 Oct 2026.