Treatment for relapsed or refractory acute myeloid leukemia

Venetoclax, Cladribine Plus Low-dose Cytarabine for Relapsed/Refractory Acute Myeloid Leukemia: a Multicenter, Randomized, Controlled Study

This study is testing a new combination treatment for people with relapsed or refractory acute myeloid leukemia to see if it helps them respond better than standard therapies.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 68 (estimated)
• Ages: 16 Years to 70 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Suzhou, Jiangsu)
• Trial ID: NCT05657639 on ClinicalTrials.gov

What this trial studies

This study investigates the efficacy and safety of a treatment regimen combining cladribine, low-dose cytarabine, and venetoclax for patients with relapsed or refractory acute myeloid leukemia (R/R AML). The CAV regimen aims to improve response rates in a patient population that typically shows resistance to standard chemotherapy. By utilizing cladribine's cytotoxic properties alongside venetoclax, the study seeks to provide a novel therapeutic option for those with limited prognosis. The trial will assess both the effectiveness and safety of this combination therapy in a Phase 2 setting.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 16-70 diagnosed with relapsed or refractory acute myeloid leukemia.

Not a fit: Patients with serious heart, lung, liver, or kidney dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for patients with relapsed or refractory acute myeloid leukemia.

How similar studies have performed: While the efficacy of cladribine combined with cytarabine and venetoclax has not been previously reported, other studies have shown promise with similar combinations in treating R/R AML.

Eligibility criteria

Inclusion Criteria:

1. Aged 16-70 years old.
2. Diagnosed with R/R AML.
3. Patients with AML must meet one of the following criteria, A or B: A: Refractory AML disease was defined as follows: (1) failure to attain CR following exposure to at least 2 courses of standard or intensive induction therapy; or (2) bone marrow leukemia cell decline index (BMCDI) \< 50% and \> 20% after 1 course of standard or intensive induction therapy. B: Relapsed AML disease was defined as follows:

(1) reappearance of leukemic blasts in the peripheral blood after CR; or (2) detection of ≥ 5% blasts in the BM not attributable to another cause (e.g., BM regeneration after consolidation therapy); or (3) extramedullary relapse.

4\. ECOG performance status score less than 2. 5. Expected survival time ≥12 weeks. 6. Without serious heart, lung, liver, or kidney dysfunction. 7. Able to understand and provide informed consent.

Exclusion Criteria:

1. Patients who are allergic to the study drug or drugs with similar chemical structures.
2. Pregnant or lactating women, and women of childbearing age who do not want to practice effective methods of contraception.
3. Active infection.
4. Active bleeding.
5. Patients with new thrombosis, embolism, cerebral hemorrhage, or other diseases or a medical history within one year before enrollment.
6. Patients with mental disorders or other conditions.
7. Liver function abnormalities (total bilirubin \> 1.5 times of the upper limit of the normal range, ALT/AST \> 2.5 times of the upper limit of the normal range or patients with liver involvement whose ALT/AST \> 1.5 times the upper limit of the normal range), or renal dysfunction (Ccr\<50ml/h).
8. Patients with a history of clinically significant QTc interval prolongation (male \> 450 ms; female \> 470 ms), ventricular heart tachycardia and atrial fibrillation, II-degree heart block, myocardial infarction attack within one year before enrollment, and congestive heart failure, and patients with coronary heart disease who have clinical symptoms and requiring drug treatment.
9. Patients who relapsed after allogeneic hematopoietic stem cell transplantation.
10. Drug abuse or long-term alcohol abuse that would affect the evaluation results.
11. Patients who have received organ transplants.
12. Patients not suitable for the study according to the investigator's assessment.

Where this trial is running

Suzhou, Jiangsu

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

Study contacts

• Study coordinator: Sheng-Li Xue, M. D.

Conditions Acute Myeloid Leukemia; venetoclax; cladribine; cytarabine; relapsed/refractory AML