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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-10-01

Study Completion Date

2027-10-01

Brief Summary

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

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Detailed Description

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The research team has shown that C6 ceramide nanoliposome (CNL) has anti-cancer activity in laboratory models of AML and that when it is combined with other cancer-fighting drugs, it works better.

The primary goal of this study is to evaluate the safety of CNL given without other cancer treatments in patients with AML where either their initial treatment didn't work or it stopped working and the AML came back (refractory or relapsed AML, aka RR-AML). This study seeks to determine the right dose to start with in later studies when CNL is combined with other drugs in potential future studies.

CNL is given by intravenous (IV) infusion and will be given twice a week in this study. Participants will receive study treatment as long as it is considered safe for them to continue, though their disease status will be checked regularly to make sure that their disease has not gotten worse. Blood samples will be collected at many time-points to see how their bodies are responding to the drug and how long it stays in the blood.

The first patients in the study will start at one dose of the drug and, if that is shown to be safe, the next group will be treated at a slightly higher dose. Participants will be given CNL by intravenous (IV) infusion twice a week over about 2 hours and then they will be monitored for about 2 hours to make sure they don't have any bad side effects, but initially patients will be required to stay at the site for about 6 hours after the start of the infusion in order to get blood draws to see how long the drug stays active in their system.

Participants will have a bone marrow biopsy before their second "cycle" of drug (after about 1 month) and then again before their third cycle of drug in order to see how their disease is responding. After that, bone marrow biopsies will be about every other cycle based on what the study doctor recommends. If the doctor doesn't think that CNL is helping their disease, of if their doctor decides that it is not safe for them to continue, they will be taken off study treatment. Participants will be followed for safety and disease status for up to 6 months.

Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Single group assignment

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open Label Administration of Ceramide NanoLiposome

Ceramide NanoLiposome will be administered by Intravenous Dosing twice per week in accordance with the protocol relative to dose escalation. There is no placebo group or arm of the study.

Group Type EXPERIMENTAL

Ceramide NanoLiposome (Ceraxa)

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type DRUG

Other Intervention Names

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Ceramide NanoLiposome

Eligibility Criteria

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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) 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).
4. Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2
5. 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.
6. 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
7. QT-interval corrected according to Fridericia's formula (QTcF) \< 450 ms on one electrocardiogram (ECG) at screening

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, or have received any investigational agent within one week of registration.
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, prostate, 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 or AML with active central nervous system (CNS) involvement
8. Untreated severe (in the opinion of the treating investigator) infection
9. Active and uncontrolled infection with HIV (viral load is detectable by PCR)
10. Active infection with Hepatitis B virus (HbSAg positive or PCR with detectable viral load) or Hepatitis C virus (viral load detectable by PCR).
11. 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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No