Acute Myeloid Leukemia, in RelapsePhase 1KNAN2001NCT04716452
Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia
A Phase 1 study testing Ceramide NanoLiposome (Ceraxa) in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia, Refractory, run by Keystone Nano, Inc at 1 site in United States.
Trial summary
The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) . Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, Refractory, Refractory/Relapse Acute Myeloid Leukemia
- What is being tested?
- Ceramide NanoLiposome (Ceraxa)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2025to 28 Feb 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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) 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).
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- Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
- ECOG performance status must be ≤2
- 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.
- 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.
Who cannot join
- 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 during study treatment and not for at least within one week prior to starting study treatment.
- 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, non-metastatic prostate cancer, 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.
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- Evidence of isolated extramedullary disease.
- Acute Promyelocytic Leukemia.
- AML with active central nervous system (CNS) involvement (as determined by study investigator).
- Severe infection requiring treatment that would interfere with study drug(s) or study participation in the opinion of the treating investigator.
- 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.
- All adverse reactions from prior therapy must have recovered to Grade ≤ 1 or acceptable baseline per treating investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Bernadette M Adair, BS contact
United States1 site, 1 recruiting
- Recruiting
University of Virginia Cancer Center
Charlottesville, Virginia, 22903
Cory Caldwell, 434-297-4182, CLCALMCRCs@uvahealth.org
Michael Keng, MD
About this study
Official title: Phase I Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia (RR-AML)
The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .
Read the detailed description
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, or 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
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, Refractory, Refractory/Relapse Acute Myeloid Leukemia
- Being tested
- Ceramide NanoLiposome (Ceraxa)
- Keywords
- Acute Myeloid Leukemia, Relapsed, Refractory, Ceramide, AML, NanoLiposome, Relapsed/Refratory Acute Myleoid Leukemia, NanoLiposomes, Safety, CNL, Ceraxa
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ceramide NanoLiposome (Ceraxa)Drug
Also called Ceramide NanoLiposome
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.
| Study group | Role | What they receive |
|---|---|---|
| Open Label Administration of Ceramide NanoLiposome | Experimental | 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. Receives: Drug: Ceramide NanoLiposome (Ceraxa) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients with Dose Limiting Toxicities as defined in Protocol Section 13.5
Dose Limiting Toxicities within the first cycle of CNL monotherapy. See section 13.5 of Protocol for the complete list of Dose Limiting Toxicities
Measured: At the end of the the first cycle of administration (each cycle is 28 days)
Number of Patients with Adverse Events
Number of Patients with Adverse Events
Measured: Through study completion, an average of 24 weeks
Severity of Adverse Events
Severity of Adverse Event As Described in Protocol
Measured: Through study completion, an average of 24 weeks
Duration of Adverse Events
Duration of Adverse Events, As Described in Protocol, measured in days
Measured: Length of Adverse Events as measured in days, measured through study completion, an average of 24 weeks
Duration of therapy
Duration of therapy provided as measured in days
Measured: Through study completion, an average of 24 weeks
Dose Levels achieved during study
Dose levels administered in milligrams per m2
Measured: Through study completion, an average of 24 weeks
Concentration Max (C Max)
Maximum Serum Concentration measured, in nanograms/milliliter
Measured: Through cycle one, 28 days (each cycle is 28 days)
Time to Maximum Study Drug (T Max)
Time to maximum concentration measured, in minutes
Measured: Through cycle one, 28 days (each cycle is 28 days)
Half Life of Study Drug
Time for drug to be reduced to half of the starting concentration (in minutes)
Measured: Through cycle one, 28 days (each cycle is 28 days)
Study Drug Clearance
The Amount of Study Drug Cleared per unit time (Nanograms/Minute)
Measured: Through cycle one, 28 days (each cycle is 28 days)
Ratio of C16/C24 Ceramides
Ratio of Ceramide 16 to Ceramide 24 (ng of C16/ng of C18) from bone marrow biopsy
Measured: After one cycle of therapy (Day 28)
Clinical Response - Complete Response
Complete Response
Measured: After Cycle Two (56 days)
Clinical Response - Complete Response with Incomplete Hematologic Recovery (CRi)
Complete Response with Incomplete Hematological Recovery as defined by blasts in bone marrow
Measured: After Cycle Two (56 days)
Clinical Response - Partial Remission
Partial Remission (as defined by blasts in bone marrow)
Measured: After Cycle Two (56 days)
Secondary outcomes (5)
Number of Patients with Grade 3 or 4 Adverse Events
Grade 3 and 4 adverse events as defined by CTCAE v5.0
Measured: Through study completion, an average of 24 weeks
Overall Response
Overall response of CNL monotherapy in patients with RR-AML: Complete remission (CR) + Complete remission with incomplete count recovery (CRi) + partial response (PR). CR, CRi and PR as defined as European LeukemiaNet 2017 (ELN 2017) response criteria
Measured: Through study completion, an average of 24 weeks, and up to 24 weeks afterwards (total of 48 weeks)
Event Free Survival
Event-free survival (EFS): the time from registration until documented refractory disease, relapse after achieving CR/CRi, or death from any cause, whichever is observed first
Measured: From registration to 24 weeks after completion of experimental drug treatment
Overall Survival
Overall survival (OS)
Measured: From registration to 24 weeks following drug administration
Quality of Life according to EORTC Quality of Life Questionnaire (QLQ) C30
Quality of life according to EORTC Quality of Life Questionnaire C30
Measured: Prior to starting study treatment, on day 1 of each cycle (each cycle is 28 days), and at end of treatment (which is expected to be 2 to 3 months (cycles) for most patients
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2021
- Study start1 Oct 2025
- Primary completion30 Jan 2027 (estimated)
- Study completion28 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia, in RelapseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single group assignment
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Keystone Nano, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Virginia, Milton S. Hershey Medical Center, National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Daniel Vlock, MD, Study director, Keystone Nano, IncChristopher Prior, Ph.D., Study chair, Keystone Nano
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04716452
- Sponsor's protocol ID
- KNAN2001
- Other IDs
- 5R44CA275609-02
- Acronym
- KNAN2001
- Status last verified
- Nov 2026
- First submitted
- 30 Nov 2020
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Likely to be shared with PO1 Researchers at UVA and Penn State. In addition to yearly FDA reports, anticipate potential manuscripts as well but not yet decided with all participants.
Publications
Barth BM, Wang W, Toran PT, Fox TE, Annageldiyev C, Ondrasik RM, Keasey NR, Brown TJ, Devine VG, Sullivan EC, Cote AL, Papakotsi V, Tan SF, Shanmugavelandy SS, Deering TG, Needle DB, Stern ST, Zhu J, Liao J, Viny AD, Feith DJ, Levine RL, Wang HG, Loughran TP Jr, Sharma A, Kester M, Claxton DF. Sphingolipid metabolism determines the therapeutic efficacy of nanoliposomal ceramide in acute myeloid leukemia. Blood Adv. 2019 Sep 10;3(17):2598-2603. doi: 10.1182/bloodadvances.2018021295.
PubMed 31488436Morad SAF, MacDougall MR, Abdelmageed N, Kao LP, Feith DJ, Tan SF, Kester M, Loughran TP Jr, Wang HG, Cabot MC. Pivotal role of mitophagy in response of acute myelogenous leukemia to a ceramide-tamoxifen-containing drug regimen. Exp Cell Res. 2019 Aug 15;381(2):256-264. doi: 10.1016/j.yexcr.2019.05.021. Epub 2019 May 18.
PubMed 31112736Kester M, Bassler J, Fox TE, Carter CJ, Davidson JA, Parette MR. Preclinical development of a C6-ceramide NanoLiposome, a novel sphingolipid therapeutic. Biol Chem. 2015 Jun;396(6-7):737-47. doi: 10.1515/hsz-2015-0129.
PubMed 25838296
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of C6 Ceramide NanoLiposome (CNL) in Patients With Relapsed/Refractory Acute Myeloid Leukemia (RR-AML)
- ClinicalTrials.gov ID
- NCT04716452
- Acronym / sponsor ID
- KNAN2001 / KNAN2001
- Conditions
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, Refractory, Refractory/Relapse Acute Myeloid Leukemia
- Keywords
- Acute Myeloid Leukemia, Relapsed, Refractory, Ceramide, AML, NanoLiposome, Relapsed/Refratory Acute Myleoid Leukemia, NanoLiposomes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Keystone Nano, IncIndustry (pharma, biotech, devices)
- Collaborators
- University of Virginia, Milton S. Hershey Medical Center, National Cancer Institute (NCI)
- Study start
- 1 Oct 2025 (actual)
- Primary completion
- 30 Jan 2027 (estimated)
- Study completion
- 28 Feb 2027 (estimated)
- First posted
- 20 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study objective is to evaluate patient safety for patients with refractory and relapsed AML being treated with Ceramide NanoLiposome (CNL) .
Detailed description
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, or 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single group assignment
| Arm | Type | Receives |
|---|---|---|
| Open Label Administration of Ceramide NanoLiposome | Experimental | Drug: Ceramide NanoLiposome (Ceraxa)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 may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- 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) 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.
Key exclusion criteria (12 listed)
- 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 during study treatment and not for at least within one week prior to starting study treatment.
- 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, non-metastatic prostate cancer, 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ceramide NanoLiposome (Ceraxa)Also: Ceramide NanoLiposomeCeramide 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. | Drug | Open Label Administration of Ceramide NanoLiposome |
Primary outcome
Number of Patients with Dose Limiting Toxicities as defined in Protocol Section 13.5
Time frame: At the end of the the first cycle of administration (each cycle is 28 days)
Dose Limiting Toxicities within the first cycle of CNL monotherapy. See section 13.5 of Protocol for the complete list of Dose Limiting Toxicities
Number of Patients with Adverse Events
Time frame: Through study completion, an average of 24 weeks
Number of Patients with Adverse Events
Severity of Adverse Events
Time frame: Through study completion, an average of 24 weeks
Severity of Adverse Event As Described in Protocol
Duration of Adverse Events
Time frame: Length of Adverse Events as measured in days, measured through study completion, an average of 24 weeks
Duration of Adverse Events, As Described in Protocol, measured in days
Duration of therapy
Time frame: Through study completion, an average of 24 weeks
Duration of therapy provided as measured in days
Dose Levels achieved during study
Time frame: Through study completion, an average of 24 weeks
Dose levels administered in milligrams per m2
Concentration Max (C Max)
Time frame: Through cycle one, 28 days (each cycle is 28 days)
Maximum Serum Concentration measured, in nanograms/milliliter
Time to Maximum Study Drug (T Max)
Time frame: Through cycle one, 28 days (each cycle is 28 days)
Time to maximum concentration measured, in minutes
Half Life of Study Drug
Time frame: Through cycle one, 28 days (each cycle is 28 days)
Time for drug to be reduced to half of the starting concentration (in minutes)
Study Drug Clearance
Time frame: Through cycle one, 28 days (each cycle is 28 days)
The Amount of Study Drug Cleared per unit time (Nanograms/Minute)
Ratio of C16/C24 Ceramides
Time frame: After one cycle of therapy (Day 28)
Ratio of Ceramide 16 to Ceramide 24 (ng of C16/ng of C18) from bone marrow biopsy
Clinical Response - Complete Response
Time frame: After Cycle Two (56 days)
Complete Response
Clinical Response - Complete Response with Incomplete Hematologic Recovery (CRi)
Time frame: After Cycle Two (56 days)
Complete Response with Incomplete Hematological Recovery as defined by blasts in bone marrow
Clinical Response - Partial Remission
Time frame: After Cycle Two (56 days)
Partial Remission (as defined by blasts in bone marrow)
5 secondary outcomes
Number of Patients with Grade 3 or 4 Adverse Events
Time frame: Through study completion, an average of 24 weeks
Overall Response
Time frame: Through study completion, an average of 24 weeks, and up to 24 weeks afterwards (total of 48 weeks)
Event Free Survival
Time frame: From registration to 24 weeks after completion of experimental drug treatment
Overall Survival
Time frame: From registration to 24 weeks following drug administration
Quality of Life according to EORTC Quality of Life Questionnaire (QLQ) C30
Time frame: Prior to starting study treatment, on day 1 of each cycle (each cycle is 28 days), and at end of treatment (which is expected to be 2 to 3 months (cycles) for most patients
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2026.
- Background: PubMed 31488436Barth BM, Wang W, Toran PT, Fox TE, Annageldiyev C, Ondrasik RM, Keasey NR, Brown TJ, Devine VG, Sullivan EC, Cote AL, Papakotsi V, Tan SF, Shanmugavelandy SS, Deering TG, Needle DB, Stern ST, Zhu J, Liao J, Viny AD, Feith DJ, Levine RL, Wang HG, Loughran TP Jr, Sharma A, Kester M, Claxton DF. Sphingolipid metabolism determines the therapeutic efficacy of nanoliposomal ceramide in acute myeloid leukemia. Blood Adv. 2019 Sep 10;3(17):2598-2603. doi: 10.1182/bloodadvances.2018021295.
- Background: PubMed 31112736Morad SAF, MacDougall MR, Abdelmageed N, Kao LP, Feith DJ, Tan SF, Kester M, Loughran TP Jr, Wang HG, Cabot MC. Pivotal role of mitophagy in response of acute myelogenous leukemia to a ceramide-tamoxifen-containing drug regimen. Exp Cell Res. 2019 Aug 15;381(2):256-264. doi: 10.1016/j.yexcr.2019.05.021. Epub 2019 May 18.
- Background: PubMed 25838296Kester M, Bassler J, Fox TE, Carter CJ, Davidson JA, Parette MR. Preclinical development of a C6-ceramide NanoLiposome, a novel sphingolipid therapeutic. Biol Chem. 2015 Jun;396(6-7):737-47. doi: 10.1515/hsz-2015-0129.
- Individual participant dataSharing planned: Yes. Likely to be shared with PO1 Researchers at UVA and Penn State. In addition to yearly FDA reports, anticipate potential manuscripts as well but not yet decided with all participants.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Bernadette M Adair, BS contact
Daniel Vlock, MD study director
Keystone Nano, Inc
Christopher Prior, Ph.D. study chair
Keystone Nano
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ceramide NanoLiposome (Ceraxa) in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia, Refractory, run by Keystone Nano, Inc at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact James H Adair, Ph.D. (814-360-4654, jadair@pendreabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients with Dose Limiting Toxicities as defined in Protocol Section 13.5 (time frame: At the end of the the first cycle of administration (each cycle is 28 days)). The study lists 14 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04716452, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.