VA-CIG as initial intensive therapy for fit adults with acute myeloid leukemia

A Multicenter, Prospective, Single-Arm Clinical Trial of Venetoclax in Combination With Azacitidine, Cytarabine, Idarubicin and G-CSF (VA-CIG) for Patients With Previously Untreated Acute Myeloid Leukemia

This study will test whether combining venetoclax with azacitidine, idarubicin, low-dose cytarabine, and G-CSF works as induction treatment for adults aged 18–70 with previously untreated, fit AML.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Beijing 302 Hospital (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Beijing)
• Trial ID: NCT07514832 on ClinicalTrials.gov

What this trial studies

This is a prospective, multicenter, single-arm Phase 2 trial testing the VA-CIG induction regimen (venetoclax + azacitidine + idarubicin + low-dose cytarabine + G-CSF) in patients with newly diagnosed AML who are eligible for intensive chemotherapy. Eligible patients are 18–70 years old with ECOG performance status ≤2 and no prior AML-directed therapy, and they receive the combination regimen with planned monitoring for response and toxicity. Primary outcomes focus on remission rates and safety, with secondary measures including survival endpoints compared against historical controls. The single-arm design means there is no randomized comparator and results will inform whether larger controlled trials are warranted.

Who should consider this trial

Good fit: Ideal candidates are adults 18–70 with newly diagnosed (non-APL) AML who are judged fit for intensive chemotherapy (ECOG ≤2) with adequate organ function and no prior AML treatment.

Not a fit: Patients older than 70, those unfit for intensive chemotherapy, those with acute promyelocytic leukemia, or those with significant organ dysfunction are unlikely to benefit from this intensive regimen.

Why it matters

Potential benefit: If successful, the VA-CIG regimen could increase remission rates and provide a more effective induction option for fit adults with newly diagnosed AML.

How similar studies have performed: Venetoclax plus hypomethylating agents has shown strong activity in older or unfit AML, and early clinical work combining venetoclax with intensive chemotherapy has produced promising response rates but remains investigational.

Eligibility criteria

Inclusion Criteria:

* Diagnosis of acute myeloid leukemia (AML, excluding acute promyelocytic leukemia) confirmed by morphology, immunophenotyping and molecular genetics, in accordance with the WHO 2022 diagnostic criteria for AML;
* Age 18-70 years, with no gender restriction;
* No prior AML-related treatment has been received; exceptions are made for the use of hydroxyurea or similar agents during the diagnostic screening phase to control peripheral blood leukemic blasts;
* Patients must be assessed as tolerable to intensive chemotherapy regimens; evaluation of tolerance to intensive chemotherapy shall be performed in accordance with the Ferrara 2013 criteria (Appendix A);
* Eastern Cooperative Oncology Group (ECOG) performance status score ≤ 2;
* Serum creatinine ≤ 2.0 × upper limit of normal (ULN), or creatinine clearance \> 40 mL/min calculated by the Cockcroft-Gault formula for glomerular filtration rate (GFR);
* Total bilirubin ≤ 2 × ULN, and aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤ 3 × ULN;
* Left ventricular ejection fraction (LVEF) ≥ 45%, or LVEF measured by echocardiography (ECHO) within the normal range;
* Expected survival \> 3 months.

Exclusion Criteria:

* Subjects with a history of myeloproliferative neoplasms (MPNs), including myelofibrosis, essential thrombocythemia, polycythemia vera, chronic myeloid leukemia (CML) with or without BCR-ABL1 translocation, and acute myeloid leukemia (AML) with BCR-ABL1 translocation;
* Patients with a prior history of venetoclax or azacitidine (Aza) treatment for other diseases;
* Known hypersensitivity to any component of the investigational medicinal products;
* History of other concurrent malignancies within 2 years prior to enrollment, except:

Adequately treated carcinoma in situ of the cervix or breast; Basal cell carcinoma or localized squamous cell carcinoma of the skin; Previously controlled malignancies treated with radical surgical resection (or other curative modalities), etc.

* Presence of uncontrolled severe infection or active bleeding;
* Pregnant or lactating women;
* Subjects with active, treatment-uncontrolled viral infection caused by HIV, hepatitis B virus, or hepatitis C virus;
* Subjects with evidence of central nervous system leukemia before treatment initiation;
* Women of childbearing potential who do not agree to use at least one reliable contraceptive method from Day 1 of the study until 90 days after the last dose of study medication; sexually active male subjects who do not agree to use contraceptive measures from Day 1 of the study until 90 days after the last dose of study medication; male subjects who do not agree to refrain from sperm donation from the start of study drug administration until at least 90 days after the last dose;
* Subjects with epilepsy requiring pharmacotherapy, dementia, or other abnormal psychiatric conditions that impair the ability to understand or comply with the study protocol;
* Presence of psychiatric disorders or cognitive impairment that prevents cooperation with treatment and follow-up; conditions limiting oral drug intake or gastrointestinal absorption.

Where this trial is running

Beijing

• Chinese PLA General Hospital — Beijing, China (Recruiting)

Study contacts

• Study coordinator: Dr. Gao Xiaoning, Chief Physician, Professor

Conditions AML; Venetoclax; chemotherapy; BCL2 inhibitor