Clinical Trial of WBC100 Capsule in Relapsed/Refractory Acute Myeloid Leukemia

NCT07014449 | Recruiting Phase 1

• 1 other identifier: WB002
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief 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). 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.

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

Outcome Measures

Primary Outcomes (3)

• Incidence and severity of dose-limiting toxicities (DLTs)

  Evaluated according to NCI-CTCAE version 5.0.

  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.

  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.

  28 days

Secondary Outcomes (20)

• Cmax

  28 days

• Tmax

  28 days

• AUC0-t

  28 days

• AUC0-inf

  28 days

• T1/2

  28 days

Other Outcomes (1)

• Exploratory biological analysis of bone marrow aspirate samples

  Cycle 1 (28 days)

Study Arms (1)

WBC100 Capsules

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.

Drug: WBC100 QD

Interventions

WBC100 QD DRUG

WBC100 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.

WBC100 Capsules

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Signed informed consent and compliance with study procedures;
• \. Male or female participants aged ≥18 years at the time of consent;
• \. Diagnosis of relapsed or refractory acute myeloid leukemia (R/R AML) according to the 2016 World Health Organization (WHO) classification;
• \. ECOG PS 0-2;
• \. Life expectancy ≥3 months;
• \. Adequate bone marrow reserve and organ function as defined below:
• 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.
• \. 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.

You may not qualify if:

• \. Known hypersensitivity to WBC100 capsules or any of their excipients;
• \. Diagnosis of acute promyelocytic leukemia (APL);
• \. Diagnosis of mixed phenotype acute leukemia, chronic myeloid leukemia in blast crisis, or AML transformed from myelodysplastic syndromes (MDS) or myeloproliferative neoplasms (MPN);
• \. Subjects with relapse after allogeneic HSCT, grade ≥ 2 acute GVHD, extensive chronic GVHD requiring immunosuppressive therapy, or autologous HSCT within the past 90 days;
• \. Subjects who have undergone major surgery, have active ulcers, or have unhealed wounds within 28 days prior to the first dose;
• \. 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;
• \. 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).
• \. 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;
• \. History or presence of immunodeficiency, autoimmune disease requiring systemic immunosuppressants, or organ transplantation;
• \. 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;
• \. Known risk factors for QT prolongation, including congenital long QT syndrome or drug-induced arrhythmia history;

Sponsors & Collaborators

• Hangzhou Weben Pharma Co., Ltd: lead

Study Sites (1)

The First Affiliated Hospital, School of Medicine, Zhejiang University

Hangzhou, Zhejiang, 310003, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Hematologic Neoplasms; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplasms by Site; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Biao Zhang, Ph.D.

CONTACT

+86 13989822331; bzhang@webenpharma.com

Baorui Kong, M.Med.

CONTACT

+86 17335563516; brkond@webenpharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2025
• First Posted: June 11, 2025
• Study Start: November 3, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: June 11, 2025

Record last verified: 2025-06

Locations

CN (1)