Ceramide NanoLiposome (Ceraxa): Ceramide 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.
Study summary
The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Signed informed consent is obtained prior to conducting any study-specific screening procedures.
2. Willing and able to understand the nature of this study and to comply with the study and follow-up procedures.
3. 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).
4. Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
5. ECOG performance status must be ≤2
6. 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.
7. 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.
Exclusion Criteria:
Patients meeting any of the following criteria are ineligible for study entry:
1. 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.
2. 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.
3. Since the teratogenic potential of this combination is currently unknown, females who are pregnant or lactating are excluded.
4. 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.
5. 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.
6. Evidence of isolated extramedullary disease.
7. Acute Promyelocytic Leukemia.
8. AML with active central nervous system (CNS) involvement (as determined by study investigator).
9. Severe infection requiring treatment that would interfere with study drug(s) or study participation in the opinion of the treating investigator.
10. 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.
11. All adverse reactions from prior therapy must have recovered to Grade ≤ 1 or acceptable baseline per treating investigator.
Primary outcome measure(s)
Number of Patients with Dose Limiting Toxicities as defined in Protocol Section 13.5 — At the end of the the first cycle of administration (each cycle is 28 days) 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 — Through study completion, an average of 24 weeks Number of Patients with Adverse Events
Severity of Adverse Events — Through study completion, an average of 24 weeks Severity of Adverse Event As Described in Protocol
Duration of Adverse Events — Length of Adverse Events as measured in days, measured through study completion, an average of 24 weeks Duration of Adverse Events, As Described in Protocol, measured in days
Duration of therapy — Through study completion, an average of 24 weeks Duration of therapy provided as measured in days
Dose Levels achieved during study — Through study completion, an average of 24 weeks Dose levels administered in milligrams per m2
Concentration Max (C Max) — Through cycle one, 28 days (each cycle is 28 days) Maximum Serum Concentration measured, in nanograms/milliliter
Time to Maximum Study Drug (T Max) — Through cycle one, 28 days (each cycle is 28 days) Time to maximum concentration measured, in minutes
Half Life of Study Drug — Through cycle one, 28 days (each cycle is 28 days) Time for drug to be reduced to half of the starting concentration (in minutes)
Study Drug Clearance — Through cycle one, 28 days (each cycle is 28 days) The Amount of Study Drug Cleared per unit time (Nanograms/Minute)
Ratio of C16/C24 Ceramides — After one cycle of therapy (Day 28) Ratio of Ceramide 16 to Ceramide 24 (ng of C16/ng of C18) from bone marrow biopsy
Clinical Response - Complete Response — After Cycle Two (56 days) Complete Response
Clinical Response - Complete Response with Incomplete Hematologic Recovery (CRi) — After Cycle Two (56 days) Complete Response with Incomplete Hematological Recovery as defined by blasts in bone marrow
Clinical Response - Partial Remission — After Cycle Two (56 days) Partial Remission (as defined by blasts in bone marrow)
Trial sites (1)
Facility
City
Region
Status
University of Virginia Cancer Center
Charlottesville
Virginia
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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