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.
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;
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;
Single group
WBC100 QD
DrugWBC100 will be administered orally as capsules once daily in 28-day cycles. The dose-escalation phase follows an accelerated titration combined with the traditional '3+3' design.
Incidence and severity of dose-limiting toxicities (DLTs)
Evaluated according to NCI-CTCAE version 5.0.
Incidence and severity of treatment-emergent adverse events (TEAEs)
Includes lab abnormalities, physical exam findings, vital signs, and ECG changes.
Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)
Based on observed DLTs.
Cmax
Peak plasma concentration after one dose
Tmax
Time to peak plasma concentration after one dose
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
AUC0-inf
Area under the plasma concentration versus time curve; time range from 0 to infinity
Exploratory biological analysis of bone marrow aspirate samples
Bone marrow aspirate samples will be collected at each tumor assessment, when feasible, for exploratory biological analyses such as C-Myc protein expression.
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