Combination therapy for high-risk acute lymphoblastic leukemia before stem cell transplant

Venetoclax (VEN)+Azacytidine (AZA) Followed by Modified BUCY Conditioning Regimen for High Risk or Refractory/Relapsed Acute Lymphoblastic Leukemia Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)

Phase 2 Interventional The First Affiliated Hospital of Soochow University · NCT05809167

This study is testing a new combination treatment for people with high-risk acute lymphoblastic leukemia to see if it helps them do better before getting a stem cell transplant.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment55 (estimated)
Ages8 Years to 65 Years
SexAll
SponsorThe First Affiliated Hospital of Soochow University Academic / other
Drugs / interventionscyclophosphamide
Locations1 site (Suzhou, Jiangsu)
Trial IDNCT05809167 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of a combination treatment using venetoclax and azacytidine, followed by a modified busulfan and cyclophosphamide conditioning regimen for patients with high-risk or relapsed/refractory acute lymphoblastic leukemia (ALL) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study aims to optimize the conditioning regimen to improve patient outcomes and reduce the risk of relapse after transplantation. By targeting abnormal gene methylation and utilizing a selective BCL-2 inhibitor, the trial seeks to enhance the anti-tumor effects in leukemia cells. This is a prospective, open-label, single-center study conducted at The First Affiliated Hospital of Soochow University.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 14 to 65 years diagnosed with high-risk or relapsed/refractory acute lymphoblastic leukemia who require a bone marrow transplant.

Not a fit: Patients outside the age range of 14 to 65 years, those with uncontrolled infections, or those with contraindications to the treatment regimen may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve survival rates and reduce relapse in patients with high-risk acute lymphoblastic leukemia.

How similar studies have performed: While this approach is innovative, there have been encouraging case reports regarding the efficacy of venetoclax-based regimens in treating acute lymphoblastic leukemia.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

1. Age 14 to 65 years;
2. Diagnosis of high-risk or relapsed/refractory acute lymphoblastic leukemia at the time of transplant.
3. Must need a bone marrow transplant;
4. Must have the ability to observe the efficacy and events;
5. Patient must have ability to understand and willingness to provide written informed consent prior to participation in the study and any related procedures being performed.

Exclusion Criteria:

1. Age \<14 or \>65 years;
2. Uncontrolled bacterial, viral, fungal, or other infection before conditioning regimen;
3. Pregnant or lactating females;
4. Current participation in another clinical trial;
5. Contra-indication to one of the drug of the regimen;
6. Any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver or other organ system that may place the patient at undue risk to undergo the agents included in the conditioning regimen

Where this trial is running

Suzhou, Jiangsu

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Allogeneic Hematopoietic Stem Cell TransplantationAcute Lymphoblastic Leukemiavenetoclaxazacytidinehematopoietic stem cell transplantationacute lymphoblastic leukemia
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.