The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .
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
Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
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.
Since the teratogenic potential of this combination is currently unknown, females who are pregnant or lactating are excluded.
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.
Single group
Ceramide NanoLiposome (Ceraxa)
DrugCeramide NanoLiposome will be given by IV twice a week. The dose, which is based on body size, will be increased for the next group of patients if the first group of patients tolerates that dose and it will decrease for the next group if they do not tolerate the dose.
Number of Patients with Dose Limiting Toxicities as defined in Protocol Section 13.5
Dose Limiting Toxicities within the first cycle of CNL monotherapy. See section 13.5 of Protocol for the complete list of Dose Limiting Toxicities
Number of Patients with Adverse Events
Number of Patients with Adverse Events
Severity of Adverse Events
Severity of Adverse Event As Described in Protocol
Duration of Adverse Events
Duration of Adverse Events, As Described in Protocol, measured in days
Number of Patients with Grade 3 or 4 Adverse Events
Grade 3 and 4 adverse events as defined by CTCAE v5.0
Overall Response
Overall response of CNL monotherapy in patients with RR-AML: Complete remission (CR) + Complete remission with incomplete count recovery (CRi) + partial response (PR). CR, CRi and PR as defined as European LeukemiaNet 2017 (ELN 2017) response criteria
Event Free Survival
Event-free survival (EFS): the time from registration until documented refractory disease, relapse after achieving CR/CRi, or death from any cause, whichever is observed first
Overall Survival
Overall survival (OS)
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Keystone Nano, Inc
Lead sponsor
University of Virginia
Collaborator
Milton S. Hershey Medical Center
Collaborator
National Cancer Institute (NCI)
Collaborator
