RecruitingPhase 1NCT04716452

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

Phase I Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia (RR-AML)

Sponsor

Keystone Nano, Inc

Enrollment

15 participants

Start Date

Oct 1, 2025

Study Type

INTERVENTIONAL

Conditions

Acute Myeloid Leukemia, in Relapse Acute Myeloid Leukemia, Refractory Refractory/Relapse Acute Myeloid Leukemia

Summary

The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .

Eligibility

Min Age: 18 Years

Inclusion Criteria13

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) after one line of AML directed therapy
Relapsed AML: Patients who achieved a complete remission (CR) 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
Peripheral white blood cell (WBC) count \<30,000/µL. For cyto-reduction, hydroxyurea is allowed during screening and through Cycle 2, Day 3 to reduce WBC count to \< 30,000 µL.
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) ≤ 2.5 × ULN
Creatinine clearance \> 60 mL/min
QT-interval corrected according to Fridericia's formula (QTcF) \< 450 ms on one electrocardiogram (ECG) at screening

Exclusion Criteria12

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, or have received any investigational agent within one week of registration.
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, prostate, 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 or AML with active central nervous system (CNS) involvement
Untreated severe (in the opinion of the treating investigator) infection
Active and uncontrolled infection with HIV (viral load is detectable by PCR)
Active infection with Hepatitis B virus (HbSAg positive or PCR with detectable viral load) or Hepatitis C virus (viral load detectable by PCR).
Past Hematopoietic stem cell transplant (HSCT) with active graft vs host disease, immunosuppression other than low dose prednisone (5 mg), or calcineurin inhibitors within the 4 weeks before registration

Interventions

DRUGCeramide 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.