Combination therapy for high-risk blood cancers before stem cell transplant

Venetoclax + Azacytidine Followed by Modified BUCY Conditioning Regimen for High-risk Myelodysplastic Syndromes (MDS) and High-risk or Relapsed/Refractory Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)

This study is testing a new combination treatment for patients with high-risk blood cancers to see if it helps them do better after a stem cell transplant.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 70 (estimated)
• Ages: 8 Years to 65 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (Academic / other)
• Drugs / interventions: cyclophosphamide
• Locations: 1 site (Suzhou, Jiangsu)
• Trial ID: NCT05823714 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 myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study aims to improve patient outcomes by optimizing the conditioning regimen to reduce relapse rates after transplantation. It 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 for this study are patients aged 8 to 65 years with high-risk MDS or high-risk or relapsed/refractory AML who require a bone marrow transplant.

Not a fit: Patients who do not have high-risk MDS or AML, or those who are not eligible for a bone marrow transplant, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment approach could significantly improve survival rates and reduce relapse in patients with high-risk MDS and AML undergoing stem cell transplantation.

How similar studies have performed: Other studies have shown promising results with similar combination therapies in hematological malignancies, indicating potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Age 8 to 65 years;
2. Diseases must be MDS and AML; high-risk MDS: int-2 risk, high risk by the IPSS; IPSS-R int-risk (\> 3.5 points), high risk, very high risk; high risk,very high risk by the WPSS; high-risk or relapsed/refractory AML: (1) age≥60 years; (2) High white blood cell count at first diagnosis (WBC≥100\*10\^9/L); (3) secondary AML (previous history of MDS, myeloproliferative disease, or treatment-related AML, etc.); (4) Complicated with extramedullary leukemia, such as central nervous system leukemia, granulocytic sarcoma, hepatosenomegaly; （5）high risk factors and relapsed/refractory AML（reference 2022-AML-ELN guideline）（6）not in remission or ≥CR2 before transplantation;
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 \<8 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

• The First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)

Study contacts

• Study coordinator: Xiaowen Tang, MD

Conditions Allogeneic Hematopoietic Stem Cell Transplantation; Acute Myeloid Leukemia; Myelodysplastic Syndrome; venetoclax; azacytidine; hematopoietic stem cell transplantation; acute myeloid leukemia; myelodysplastic syndrome