NCT03150004

Efficacy and Pharmacogenomics of Cladribine Based Salvage Chemotherapy in Patients With Relapse/Refractory and Secondary Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MDS)

A Phase II Study of the Efficacy and Pharmacogenomics of Cladribine-based Salvage Chemotherapy in Patients With Relapse/Refractory and Secondary Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MDS)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Medical College of Wisconsin · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective phase II clinical study planned to be conducted at the Medical College of Wisconsin (MCW). After meeting the study criteria and enrollment, patients will be treated with a cladribine based salvage regimen and followed at periodic intervals to determine the primary and secondary objectives.

Detailed description

STUDY RATIONALE: The optimal treatment regimen for relapsed/refractory AML and high risk MDS progressing after hypomethylating agents is unknown. Although several chemotherapy options are available, there is no universally accepted regimen to date. Cladribine based salvage regimens have been frequently used at the investigators' center. However, it is uncertain to predict which patients are likely to respond to cladribine-based salvage or experience treatment-related toxicities. While studies have demonstrated that achievement of MRD negative complete remission (CR) is likely to be associated with a better overall survival (OS), there is limited prospective data evaluating the role of minimal residual disease (MRD) in the setting of relapsed/refractory disease. Through this study, the investigators aim to demonstrate the influence of achieving MRD negative CR on survival of patients with relapsed/refractory AML/high risk MDS treated with cladribine-based salvage therapy. In addition to the conventionally used predictive factors, we aim to incorporate pharmacogenomics to assess the efficacy and toxicity of therapy. PRIMARY OBJECTIVE: To determine the CR rate and achievement of MRD negativity after treatment with Cladribine based salvage chemotherapy regimen in patients with relapse/refractory AML/high risk MDS. SECONDARY OBJECTIVES: 1. To determine the progression free survival (PFS) and overall survival (OS) of patients treated with a cladribine based salvage chemotherapy regimen. 2. To study the pharmacogenomics of patients receiving a cladribine based salvage and determine its influence on survival, CR rate and MRD negativity. 3. Determination of disease- or patient-related factors that predict MRD negativity and survival with a cladribine based salvage regimen.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Cladribine, Cytarabine, Mitoxantrone, G-CSF (CLAG-M) regimen	Subjects will be started on CLAG-M regimen, which consists of the following: * Cladribine 5 mg/m\^2 IV over two hours on days 1-5; * Cytarabine 2 gm/m\^2 IV over four hours on days 1-5 * Mitoxantrone 10 mg/m\^2 IV on days 1-3; * G-CSF at a dose of 300 μg on days 0-5.
DRUG	Cladribine and Cytarabine (CLLDAC) Regimen	* Cladribine 5 mg/m\^2 IV over two hours on days 1-5; * Cytarabine 20 mg/m\^2 subcutaneous injection on days 1-10;

Timeline

• Start date: 2017-06-14
• Primary completion: 2025-03-14
• Completion: 2025-03-14
• First posted: 2017-05-11
• Last updated: 2026-03-30
• Results posted: 2026-03-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study