C6 ceramide nanoliposome (CNL) treatment for relapsed or refractory acute myeloid leukemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 1, open-label dose-escalation trial giving C6 ceramide in a nanoliposome (CNL) as a single agent to adults with relapsed or refractory AML to determine a safe dose for future combination studies. CNL is given by intravenous infusion twice weekly, with early patients observed longer after dosing and later patients observed for about two hours post-infusion. The study collects blood samples at multiple time points to measure how the drug behaves in the body and to monitor safety. Dose levels will be increased stepwise if prior doses are tolerated, and disease status will be checked regularly to ensure patient safety.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of Virginia Cancer Center
  Charlottesville, Virginia, United States Recruiting

Study contacts

• Study coordinator: James H Adair, Ph.D.
• Email: jadair@pendreabio.com
• Phone: 814-360-4654

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Combining cytokine-induced memory-like natural killer cells with atezolizumab for relapsed or refractory AML

  This trial will test whether giving cytokine-induced memory-like natural killer cells together with atezolizumab is safe and might help adults with relapsed or refractory acute myeloid leukemia.

  Recruiting at 7 sites Phase 1 Memorial Sloan Kettering Cancer Center · Basking Ridge, New Jersey and 6 other locations · NCT07011004

• Treatment with ARD103 CAR-T therapy for patients with relapsed or refractory AML or MDS

  This study is testing a new CAR-T therapy called ARD103 to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia or myelodysplastic syndrome.

  Recruiting at 3 sites Phase 1/2 ARCE Therapeutics, Inc. · Charlotte, North Carolina and 2 other locations · NCT06680752

• Framework for managing relapsed or refractory acute myeloid leukemia

  This study is trying to see how well new personalized treatments work for people in Europe with relapsed or refractory acute myeloid leukemia to help improve their care.

  Recruiting at 40 sites Istituto Romagnolo per lo Studio dei Tumori Dino Amadori IRST S.r.l. IRCCS · Aachen and 40 other locations · NCT06459024

• Investigating APL-4098 with Azacitidine for treating relapsed or refractory AML and high-risk MDS

  This study is testing a new treatment called APL-4098, alone and with azacitidine, to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia and high-risk myelodysplastic syndrome.

  Recruiting at 8 sites Phase 1 Apollo Therapeutics Ltd · Clayton, Victoria and 8 other locations · NCT06372717

• Using off-the-shelf CAR-NK cells to treat relapsed or refractory acute myeloid leukemia

  This study is testing a new treatment using specially modified immune cells to see if they can help people with relapsed or hard-to-treat acute myeloid leukemia feel better.

  Recruiting Early Phase 1 Peking University People's Hospital · Beijing, Beijing Municipality · NCT06201247

• Using CAR-T cell therapy targeting Tim-3 and CD123 for treating relapsed acute myeloid leukemia

  This study is testing a new CAR-T cell therapy that targets specific markers in people aged 18-70 with relapsed acute myeloid leukemia to see if it can safely improve their treatment outcomes.

  Recruiting Phase 1/2 Xuzhou Medical University · Xuzhou, Jiangsu · NCT06125652

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.