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Recruiting Phase 1 NCT04716452

NCT04716452 Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia

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Clinical Trial Summary
NCT ID NCT04716452
Status Recruiting
Phase Phase 1
Sponsor Keystone Nano, Inc
Condition Acute Myeloid Leukemia, in Relapse
Study Type INTERVENTIONAL
Enrollment 15 participants
Start Date 2025-10-01
Primary Completion 2026-09-30

Eligibility & Interventions

Sex All sexes
Min Age 18 Years
Max Age N/A
Study Type INTERVENTIONAL
Interventions
Ceramide NanoLiposome (Ceraxa)

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.

Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.

This trial targets 15 participants in total. It began in 2025-10-01 with a primary completion date of 2026-09-30.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

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

Eligibility Criteria

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.

Contact & Investigator

Central Contact

James H Adair, Ph.D.

✉ jadair@pendreabio.com

📞 814-360-4654

Principal Investigator

Daniel Vlock, MD

STUDY DIRECTOR

Keystone Nano, Inc

Frequently Asked Questions

Who can join the NCT04716452 clinical trial?

This trial is open to participants of all sexes, aged 18 Years or older, studying Acute Myeloid Leukemia, in Relapse. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

What phase is the NCT04716452 trial and what does that mean for participants?

Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.

Is NCT04716452 currently recruiting?

Yes, NCT04716452 is actively recruiting participants. Contact the research team at jadair@pendreabio.com for enrollment information.

Where is the NCT04716452 trial being conducted?

This trial is being conducted at Charlottesville, United States.

Who is sponsoring the NCT04716452 clinical trial?

NCT04716452 is sponsored by Keystone Nano, Inc. The principal investigator is Daniel Vlock, MD at Keystone Nano, Inc. The trial plans to enroll 15 participants.

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ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: July 2026  ·  Data Methodology