Ceramide NanoLiposome for Acute Myeloid Leukemia

(KNAN2001 Trial)

Enrolling by invitation at 2 trial locations

Overseen By Bernadette M Adair, BS

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Keystone Nano, Inc

Must not be taking: Investigational agents

Disqualifiers: Congestive heart failure, Angina, Arrhythmias, Myocardial infarction, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Ceramide NanoLiposome (Ceraxa) for individuals with acute myeloid leukemia (AML) that has not responded to other treatments or has returned after treatment. Researchers aim to determine if this treatment is safe for patients under these conditions. Participants receive the treatment through an IV twice a week, with no placebo group. It suits adults with AML who have tried other treatments without success or have experienced a relapse. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot be on other investigational agents. Hydroxyurea is allowed to manage white blood cell counts.

Is there any evidence suggesting that Ceramide NanoLiposome is likely to be safe for humans?

Research has shown that Ceramide NanoLiposome (CNL) appears safe. In studies with patients who have hard-to-treat acute myeloid leukemia (AML), the treatment was generally well-tolerated. Early signs indicated potential benefits, such as longer periods without disease progression. Tests on how the treatment moves and acts in the body also supported its safety. While more research is needed, these early findings suggest that CNL could be a safe option for those with AML that has returned or is not responding to other treatments. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for acute myeloid leukemia, which typically involve chemotherapy and targeted therapy drugs, Ceramide NanoLiposome (Ceraxa) offers a novel approach. This treatment uses a unique delivery system called a nanoliposome, allowing for the precise delivery of ceramide directly into cancer cells. Ceramide is an active ingredient that can induce cancer cell death, potentially leading to fewer side effects and improved effectiveness. Researchers are excited because this method could offer a more targeted attack on leukemia cells, possibly improving outcomes for patients.

What evidence suggests that Ceramide NanoLiposome might be an effective treatment for acute myeloid leukemia?

Research has shown that Ceramide NanoLiposome (CNL), which participants in this trial will receive, might enhance treatments for acute myeloid leukemia (AML). Studies have found that CNL can boost the effects of other cancer drugs like venetoclax and cytarabine in AML models. Research on fat-like molecules, known as sphingolipids, suggests that CNL targets specific pathways crucial for cancer cell survival. This targeting may help destroy cancer cells by disrupting these pathways. Early results are promising, but further research is needed to confirm its effectiveness in people. ^{1 6 7 8 9}

Who Is on the Research Team?

Daniel Vlock, MD

Principal Investigator

Keystone Nano, Inc

Christopher Prior, Ph.D.

Principal Investigator

Keystone Nano

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Acute Myeloid Leukemia (AML) that didn't respond to initial treatment or came back after. They should be in fairly good health otherwise, able to follow the study plan, and not have had a stem cell transplant recently or other cancers within the last year.

Inclusion Criteria

Creatinine clearance > 60 mL/min

QT-interval corrected according to Fridericia's formula (QTcF) < 450 ms on one electrocardiogram (ECG) at screening

Total bilirubin ≤ 1.5 × upper limit of normal (ULN) or < 3 x ULN for patients with Gilbert-Meulengracht Syndrome

Exclusion Criteria

Patients may not be receiving any other concurrent investigational agents, or have received any investigational agent within one week of registration.

My leukemia affects my brain or spinal cord.

I do not have any severe illnesses that my current medications cannot control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive C6 Ceramide NanoLiposome (CNL) by intravenous infusion twice a week. Dose escalation is based on safety, and participants are monitored for adverse effects.

24 weeks

Twice weekly visits for infusion

Follow-up

Participants are monitored for safety and disease status for up to 6 months after treatment completion.

24 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ceramide NanoLiposome (Ceraxa)

Trial Overview The trial tests Ceramide NanoLiposome (Ceraxa), which might boost the effectiveness of standard cancer drugs against AML. Participants will receive CNL combined with conventional treatments to see if this improves outcomes.

How Is the Trial Designed?

Group I: Open Label Administration of Ceramide NanoLiposome Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Keystone Nano, Inc

Lead Sponsor

Trials

2

Recruited

30+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

University of Virginia

Collaborator

Trials

802

Recruited

1,342,000+

Memorial Sloan Kettering Cancer Center

Collaborator

Trials

1,998

Recruited

602,000+

Milton S. Hershey Medical Center

Collaborator

Trials

515

Recruited

2,873,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9544744/

Ceramide nanoliposomes augment the efficacy of ... Ceramide nanoliposomes augment the efficacy of venetoclax and cytarabine in models of acute myeloid leukemia.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04716452

Study of C6 Ceramide NanoLiposome (CNL) in Patients ... The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) . Detailed ...

3. reporter.nih.gov reporter.nih.gov/search/10EECF0E478CC1DE7598B8961CAA4A01A2FFCEB861BF/project-details/10704116

Ceraxa (Ceramide NanoLiposome) and Vinblastine For ... These patients have a three-year survival rate of only ten percent, and new therapies are urgently needed. The Ceraxa/Vinblastine combination is novel, never ...

4. ashpublications.org ashpublications.org/bloodadvances/article/3/17/2598/261357/Sphingolipid-metabolism-determines-the-therapeutic

Sphingolipid metabolism determines the therapeutic efficacy ... We studied sphingolipid biology in different forms of AML and evaluated the anti-AML efficacy of nanoliposomal C6-ceramide (Lip-C6).

5. grantome.com grantome.com/grant/NIH/P01-CA171983-06A1-6298

Clinical Trial of Ceramide nanoLiposomes in AML Acute Myeloid Leukemia (AML) is the 2nd most common type of leukemia diagnosed with a 5-year survival rate of only 27%. Though dose-intensive induction and ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10796569/

A phase I study of the ceramide nanoliposome in patients with ... CNL exhibited an encouraging safety profile and pharmacokinetic parameters, with some signals of efficacy including prolonged stable disease in ...

7. mycancergenome.org mycancergenome.org/content/clinical_trials/NCT04716452/

Clinical Trial: NCT04716452 The research team has shown that C6 ceramide nanoliposome (CNL) has anti-cancer activity in laboratory models of AML and that when it is ...

8. faseb.onlinelibrary.wiley.com faseb.onlinelibrary.wiley.com/doi/full/10.1096/fj.202200765R

Ceramide nanoliposomes augment the efficacy of venetoclax ... Ceramide nanoliposomes augment the efficacy of venetoclax and cytarabine in models of acute myeloid leukemia.

9. ctv.veeva.com ctv.veeva.com/study/study-of-c6-ceramide-nanoliposome-cnl-in-patients-with-relapsed-refractory-acute-myeloid-leukemia

Study of C6 Ceramide NanoLiposome (CNL) in Patients With ... The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .