NCT04716452 · Keystone Nano, Inc
Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia
(KNAN2001)
What this study is about
The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .
View original scientific description
The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .
Interventions
DRUG
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.
Primary outcome measures
Number of Patients with Dose Limiting Toxicities as defined in Protocol Section 13.5
Time frame: 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
Time frame: Through study completion, an average of 24 weeks
Number of Patients with Adverse Events
Severity of Adverse Events
Time frame: Through study completion, an average of 24 weeks
Severity of Adverse Event As Described in Protocol
Duration of Adverse Events
Time frame: 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
Time frame: Through study completion, an average of 24 weeks
Duration of therapy provided as measured in days
Dose Levels achieved during study
Time frame: Through study completion, an average of 24 weeks
Dose levels administered in milligrams per m2
Concentration Max (C Max)
Time frame: Through cycle one, 28 days (each cycle is 28 days)
Maximum Serum Concentration measured, in nanograms/milliliter
Time to Maximum Study Drug (T Max)
Time frame: Through cycle one, 28 days (each cycle is 28 days)
Time to maximum concentration measured, in minutes
Half Life of Study Drug
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: After Cycle Two (56 days)
Complete Response
Clinical Response - Complete Response with Incomplete Hematologic Recovery (CRi)
Time frame: After Cycle Two (56 days)
Complete Response with Incomplete Hematological Recovery as defined by blasts in bone marrow
Clinical Response - Partial Remission
Time frame: After Cycle Two (56 days)
Partial Remission (as defined by blasts in bone marrow)
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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.
Exclusion criteria
- Patients meeting any of the following criteria are ineligible for study entry:
- 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.
- 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.
- Acute Promyelocytic Leukemia.
- AML with active central nervous system (CNS) involvement (as determined by study investigator).
- Severe infection requiring treatment that would interfere with study drug(s) or study participation in the opinion of the treating investigator.
- 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.
Where
- Charlottesville, Virginia
Collaborators
University of Virginia, Milton S. Hershey Medical Center, National Cancer Institute (NCI)
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 31, 2026 · Source of record for eligibility and locations