Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Prior treatment

• Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmias not well controlled with medication, myocardial infarction within the previous 6 months before registration, or psychiatric illness/social situations that would limit compliance with study requirements.
• Patients may not be receiving any other concurrent investigational agents during study treatment and not for at least within one week prior to starting study treatment.
• History of any other malignancies within the preceding 12 months before registration with the exception of in-situ cancer, non-muscle invasive bladder cancer, non-metastatic prostate cancer, basal or squamous cell skin cancer.
• Past Hematopoietic stem cell transplant (HSCT) with graft vs host disease, immunosuppression other than low dose prednisone (10 mg) (or equivalent does of another immunosuppressant) within the 4 weeks before registration.
• All adverse reactions from prior therapy must have recovered to Grade ≤ 1 or acceptable baseline per treating investigator.

Other illness

• Life-threatening illnesses other than AML, uncontrolled medical conditions or organ system dysfunction that, in the Investigator's opinion, could compromise the patient's safety or put the study outcomes at risk.
• Evidence of isolated extramedullary disease.
• Severe infection requiring treatment that would interfere with study drug(s) or study participation in the opinion of the treating investigator.

Other

• Patients meeting any of the following criteria are ineligible for study entry:
• Since the teratogenic potential of this combination is currently unknown, females who are pregnant or lactating are excluded.
• Acute Promyelocytic Leukemia.
• AML with active central nervous system (CNS) involvement (as determined by study investigator).

• Signed informed consent is obtained prior to conducting any study-specific screening procedures.
• Willing and able to understand the nature of this study and to comply with the study and follow-up procedures.
• Age and Disease: ≥ 18 years of age with refractory or relapsed AML
  • Refractory AML: Patients who fail to achieve a complete remission (CR) or a complete remission with incomplete count recovery (CRi) after one or more ines of AML directed therapy.
  • Relapsed AML: Patients who achieved a complete remission (CR) or a complete remission with incomplete count recovery (CRi) with one or more prior lines of AML directed therapy but then developed a relapse of AML.
  • Note: Patients are eligible even if they have not received intensive induction chemotherapy but have been treated with other AML directed therapy like hypomethylating agents (azacitidine, decitabine).
• Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
• ECOG performance status must be ≤2
• Peripheral white blood cell (WBC) count \<30,000/µL. For cyto-reduction, the following are allowed to reduce WBC count to \< 30,000/µL:
  • hydroxyurea is allowed during screening and through the end of Cycle,
  • cytarabine is allowed during screening but not after registration and should be limited 1 g/m2 or less from time of consent to registration.
• Adequate organ function as evidenced by the following laboratory findings:
  • Total bilirubin ≤ 1.5 × upper limit of normal (ULN) or \< 3 x ULN for patients with Gilbert-Meulengracht Syndrome
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × ULN if not attributed to leukemia, or ≤ 5 x ULN if attributed to leukemia
  • Creatinine clearance \> 60 mL/min.