Efficacy and Safety of VAH as a Bridging Regimen to Allo-HCT in Relapsed/Refractory AML

NCT07091006 | Not Yet Recruiting Phase 2

• 1 other identifier: RJ-BMT-013
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

Brief Summary

Allogeneic hematopoietic cell transplantation (allo-HCT) is the only treatment that offers a possible cure for relapsed/refractory AML. Currently, the optimal preallo-HCT bridging regimen for relapsed/refractory AML patients is unclear. Venetoclax-based regimens, including Venetoclax + demethylating agents (HMA) , Venetoclax + HMA + other drugs and Venetoclax-based multidrug combinations as a bridging regimen improves response rate and post-transplant survival in relapsed/refractory AML patients. Therefore, the investigators conduct a prospective single-centre clinical study to evaluate the efficacy and safety of VAH as a transplant bridging regimen for relapsed/refractory AML.

Conditions

Relapse Leukemia

Outcome Measures

Primary Outcomes (1)

• 2-year post-transplant relapse rate

  relapse

  through study completion, an average of 2 year

Secondary Outcomes (2)

• 2-year event-free survival after transplantation

  through study completion, an average of 2 year

• Overall survival at 2 years after transplantation

  through study completion, an average of 2 year

Study Arms (1)

Experimental arm

EXPERIMENTAL

Patients with relapsed/refractory acute myeloid leukaemia eligible for enrolment should be bridged with venetoclax, azacitidine, in combination with homoharringtonine before allo-HCT.

Drug: VAH

Interventions

VAH DRUG

For R/R AML patients, VAH bridging to conditioning regimen for allo-HCT.

Experimental arm

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 14 to 70 years (inclusive), regardless of gender;
• The diagnosis of AML was confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
• Relapsed AML: After achieving complete remission (CR), the reappearance of leukemic cells in peripheral blood, or ≥5% blasts in bone marrow (excluding other causes such as bone marrow regeneration after consolidation chemotherapy), or extramedullary infiltration by leukemic cells.
• Refractory AML: Newly diagnosed cases that are unresponsive after two courses of standard induction therapy; patients who relapse within 12 months after consolidation/intensification therapy post-CR; patients who relapse after 12 months but fail to respond to conventional chemotherapy; patients with two or more relapses; or those with persistent extramedullary leukemia.
• With RUNX1::RUNX1T1 AML: Positive measurable residual disease (MRD) on bone marrow flow evaluation after the second consolidation therapy and/or less than a 3-log decrease in the RUNX1::RUNX1T1 fusion gene and diagnostic baseline values;
• ECOG score ≤2; HCT-CI score \<3; Aspartate aminotransferase (AST) ≤ 3 times ULN (upper limit of normal, ULN); Alanine aminotransferase (ALT) ≤ 3x ULN; Total serum bilirubin ≤ 1.5 times the upper limit of normal ULN unless the patient has documented Gilbert syndrome; patients with Gilbert-Meulengracht syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included; Serum creatinine ≤ 1.5 times ULN or creatinine clearance ≥ 60 mL/min; Coagulation function: International Normalised Ratio (INR) ≤ 1.5 x ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5 x ULN;
• Left ventricular ejection fraction (LVEF) ≥50%;

You may not qualify if:

• Allergies or contraindications to any of the drugs in the protocol;
• Currently have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
• Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
• Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
• Patients with neurological or psychiatric disorders;
• The patient is pregnant or breastfeeding;
• Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form.
• Other conditions that, in the opinion of the investigator, make the patient otherwise unsuitable for participation in this study;

Sponsors & Collaborators

• Ruijin Hospital: lead

Study Sites (1)

Ruijin Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, Shanghai Municipality, China

Location

Study Officials

• Xiaoxia HU

  Ruijin Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoxia HU

CONTACT

02164370045; hu_xiaoxia@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 7, 2025
• First Posted: July 29, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): May 1, 2028
• Last Updated: July 29, 2025

Record last verified: 2025-07

Locations

CN (1)