Venetoclax-containing Therapy Combined With Microtransplant for Newly Diagnosed AML

NCT ID: NCT07078591

Last Updated: 2025-07-28

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-20
• Study Completion Date: 2031-06-30

Brief Summary

The purpose of this study is to evaluate whether HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cell (GPBMC) infusion with venetoclax-containing regimens (microtransplant, MST) could improve survival in adult patients with newly diagnosed acute myeloid leukemia (AML).

Conditions

Acute Myeloid Leukemia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Participants fit for intensive therapy

This cohort will include patients fit for intensive therapy.

Induction phase:

Genetically low-risk patients Venetoclax 100mg on Day 4, 200mg on Day 5, 400mg on Days 6-11 orally; Daunorubicin 60mg/m2 or idarubicin 12mg/m2 IV daily on Days 1-3; Cytarabine 100mg/m2 IV daily on Days 1-7. No more than two cycles. Continue to consolidation phase if achieving CR/CRi.

Genetically intermediate/high-risk patients Venetoclax 100mg on Day 1, 200mg on Day 2, 400 mg on Days 3-28 orally; Azacitidine 75mg/m2 SC daily on Days 1-7. 28 days per cycle, and no more than two cycles. Continue to consolidation phase if achieving CR/CRi and refuse allogeneic HSCT.

Consolidation phase:

Patients < 60 years Cytarabine 1.5g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.

Patients ≥ 60 years Cytarabine 1g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.

Group Type: EXPERIMENTAL

Venetoclax

Intervention Type: DRUG

Given PO

Azacitidine (AZA)

Intervention Type: DRUG

Given SC

Daunorubicin

Intervention Type: DRUG

Given IV

Idarubicin（IDA）

Intervention Type: DRUG

Given IV

Cytarabine (Ara-C)

Intervention Type: DRUG

Given IV

GPBMC infusion

Intervention Type: BIOLOGICAL

HLA-mismatched donor GPBMC infusion

Participants unfit for intensive therapy but fit for lower intensity therapy

This cohort will include patients unfit for intensive therapy but fit for lower intensity therapy at diagnosis according to Ferrara 2013 criteria.

Induction phase:

Venetoclax 100mg on Day 1, 200mg on Day 2, 400 mg on Days 3-28 orally; Azacitidine 75mg/m2 SC daily on Days 1-7. 28 days per cycle, and no more than two cycles. Re-evaluate unfitness and continue to consolidation phase if achieving CR/CRi and refuse allogeneic HSCT.

Consolidation phase:

Convert to "fit" patients and < 60 years Cytarabine 1.5g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.

Convert to "fit" patients and ≥ 60 years Cytarabine 1g/m2 IV every 12 hours on Days 1, 2, 3; Venetoclax 400mg on Days 1-7. Infuse donor GPBMCs on Day 8. Totally 3-4 cycles.

Still "unfit" patients Venetoclax 400 mg on Days 1-14 orally; Azacitidine 75mg/m2 SC daily on Days 1-7. Infuse donor GPBMCs on Day 15. 28 days per cycle until progression or untolerated.

Group Type: EXPERIMENTAL

Venetoclax

Intervention Type: DRUG

Given PO

Azacitidine (AZA)

Intervention Type: DRUG

Given SC

Cytarabine (Ara-C)

Intervention Type: DRUG

Given IV

GPBMC infusion

Intervention Type: BIOLOGICAL

HLA-mismatched donor GPBMC infusion

Interventions

Venetoclax

Given PO

Intervention Type: DRUG

Azacitidine (AZA)

Given SC

Intervention Type: DRUG

Daunorubicin

Given IV

Intervention Type: DRUG

Idarubicin（IDA）

Given IV

Intervention Type: DRUG

Cytarabine (Ara-C)

Given IV

Intervention Type: DRUG

GPBMC infusion

HLA-mismatched donor GPBMC infusion

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Age >=18 years, male or female, non-limited by race or ethnicity.
• No prior anti-acute leukemia treatment (including hypomethylators for leukemia or MDS) with the exception that prior hydroxyurea and/or leukapheresis are permitted.
• Confirmed acute myeloid leukemia in accordance with WHO criteria with a WBC count < 25 × 109/L.
• Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )<= 3 × upper limit of normal(ULN), and total bilirubin <= 1.5 × ULN.
• Adequate renal function including serum creatinine <= 2 × ULN or CrCl>= 40mL/min.
• LVEF measured by echocardiogram is within the normal range (LVEF > 50%).
• The subject must have one donor who is >= 18 years old and HLA matched at 0-7/10 loci (i.e., at least 3 HLA loci must be mismatched). In addition, the donor voluntarily donates hematopoietic stem cells and signs the consent form.
• Each subject (or his/her legal representatives) must sign the Informed Consent Form (ICF), indicating that he/she understands the purpose and procedures of research, and is willing to participate in research.

Exclusion Criteria

• Acute promyelocytic leukemia, myeloid sarcoma, chronic myeloid leukemia accelerated phase and blast crisis.
• Uncontrolled infection or hemorrhage.
• Cardiovascular disease with clinical significance, such as uncontrolled or highly symptomatic cardiac arrhythmias, congestive heart failure, or myocardial infarction within 6 months prior to screening, or New York Heart Association (NYHA) function class 3 (moderate) or class 4 (severe) heart disease.
• Uncontrolled autoimmune disease or requiring immunosuppression treatment.
• History of severe blood infusion reaction.
• Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception.
• Psychiatric disorder or cognitive impairment that in the researcher's judgment would make the subject not likely to adhere to the protocol requirements.
• Any major surgery within 4 weeks before enrollment.
• Life-threatening illness other than AML or uncontrolled intercurrent illness.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Beijing 302 Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bo Cai, MD

Role: PRINCIPAL_INVESTIGATOR

Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital

Locations

Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital

Beijing, , China

Site Status: RECRUITING

Countries

China

Central Contacts

Bo Cai, MD

Role: CONTACT

caibo2008@163.com

+861066947168

Yangyang Lei, MD

Role: CONTACT

942056176@qq.com

+861066947180

Facility Contacts

Bo Cai, MD

Role: primary

caibo2008@163.com

+861066947168

Yangyang Lei, MD

Role: backup

942056176@qq.com

+861066947180

Other Identifiers

VEN-MST for AML

Identifier Type: -

Identifier Source: org_study_id