Acute Myeloid LeukemiaPhase 1ADVANCE-01NCT07905027
A Study of CLEC2A CAR T for Pediatric and Adult Patients With Relapsed or Refractory AML
A Phase 1 study testing Biological SC-CAR.CLEC2Av1 in people with Acute Myeloid Leukemia and Acute Myeloid Leukaemia Recurrent, run by Seattle Children's Hospital at 1 site in United States.
Trial summary
This is a phase I, open-label, non-randomized clinical trial that will enroll pediatric and adult research participants with CLEC2A+ acute myeloid leukemia (AML) that does not go away with treatment (is refractory) or comes back after treatment (is recurrent). Read more about this study
- Who is it for?
- Ages Up to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Myeloid Leukaemia Recurrent, Acute Myeloid Leukaemia Refractory
- What is being tested?
- Biological SC-CAR.CLEC2Av1
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2026to Feb 2043
- 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 Up to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject age ≤ 70 years
- Weight ≥ 7 kilograms
- AML that expresses CLEC2A by flow cytometry as assessed by Hematologics, Inc. Laboratory and meets one of the below definitions:
- for subjects who have previously received an allogeneic HCT, any evidence of AML post HCT detectable by flow cytometry.
- first or greater relapse of AML.
- refractory AML, defined as ≥ 0.1% leukemic cells determined by flow cytometry or >1% on biopsy after 2 cycles of chemotherapy.
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- Able to tolerate apheresis
- Life expectancy ≥ 8 weeks
- Has an appropriate stem cell donor source identified
- Lansky performance status score of ≥ 50 for subjects <16 years of age or Karnofsky score ≥ 50 for subjects ≥ 16 years. Subjects who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for purposes of assessing performance status
- The subject must discontinue all anticancer agents and radiotherapy and, in the opinion of the investigator, have fully recovered from significant acute toxic effects of all prior chemotherapy, immunotherapy, and radiotherapy:
- Chemotherapy and biologic agents: All chemotherapy and biologic therapy not specifically mentioned below must be discontinued ≥ 14 days prior to enrollment, with the exception of intrathecal chemotherapy for which there is not a required washout period.
- Steroid use: All corticosteroid therapy (unless physiologic replacement dosing) must be discontinued ≥ 7 days prior to enrollment, unless being used to treat GVHD (if being used to treat GVHD see Section 6.4.2 for requirements).
- Tyrosine Kinase Inhibitor (TKI) use: All TKIs must be discontinued ≥ 3 days prior to enrollment.
- Hydroxyurea: must be discontinued ≥ 1 day prior to enrollment.
- Gene Modified cellular therapy:
- Must be at least 30 days from most recent gene modified cell therapy infusion and document no evidence of modified cells in the peripheral blood OR
- Must be at least 60 days from most recent gene modified cell therapy.
- Adequate organ function as indicated by:
- Renal: Serum creatinine ≤ 1.5 X the upper limit of normal (ULN) based on the following: Age: 1 to < 2 yrs: Male: 0.6 mg/dL. Female: 0.6 mg/dL Age: 2 to < 6 yrs: Male: 0.8 mg/dL. Female: 0.8 mg/dL Age: 6 to <10 yrs: Male: 1 mg/dL. Female: 1 mg/dL Age: 10 to < 13 yrs: Male: 1.2 mg/dL. Female: 1.2 mg/dL Age: 13 to < 16 yrs: Male: 1.5 mg/dL. Female: 1.4 mg/dL Age: ≥ 16 yrs Male: 1.7 mg/dL. Female: 1.4 mg/dL
- Hepatic: Total bilirubin ≤ 3 times ULN for age OR conjugated bilirubin ≤ 2 mg/dL AND ALT (SGPT) ≤ 5 times ULN.
- Cardiac: Shortening fraction ≥ 28% OR ejection fraction (EF) ≥ 50% as measured by echocardiogram.
- Respiratory: Oxygen saturation ≥ 92% on room air without supplemental oxygen or mechanical ventilation.
- Laboratory values meet the following criteria:
- Absolute Lymphocyte Count (ALC) ≥ 100 cells/uL. b) Virology Testing negative within 3 months prior to enrollment, to include: i. HIV antigen \& antibody. ii. Hepatitis B surface antigen. iii. Hepatitis C antibody OR if positive, Hepatitis C PCR is negative.
- If subject is of childbearing or child-fathering potential, must agree to use highly effective contraception (Appendix 7) from the time of initial consent through 12 months following the infusion of investigational product on this trial.
- Subject and/or legally authorized representative has signed the Informed Consent Form for this study.
Who cannot join
- Active malignancy other than acute myeloid leukemia.
- Treated localized breast cancer, breast cancer receiving endocrine therapy alone, non-melanoma skin cancer, localized prostate cancer, prostate cancer receiving endocrine therapy or being managed with observation all will be allowed.
- History of symptomatic non-AML CNS disease or ongoing symptomatic CNS disease requiring medical intervention, including paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injury, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder (subjects with non-febrile seizure disorder controlled on anti-epileptic medication and without seizure activity within 1 month are eligible).
- CNS AML involvement that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and T cell infusion.
- If history of allogeneic stem cell transplant: active GVHD or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks prior to enrollment.
- If history of allogeneic stem cell transplant and patient has received donor lymphocyte infusion (DLI) the subject is <8 weeks from DLI infusion.
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- Presence of active severe infection, defined as:
- Positive blood culture within 48 hours of enrollment, OR
- Fever above 38.2° C, AND clinical signs of infection within 48 hours of enrollment.
- Primary immunodeficiency syndrome.
- Subject has received prior virotherapy.
- Subject and/or legally authorized representative unwilling to provide consent/assent for participation in the 15-year follow-up period, required if SC-CAR.CLEC2Av1 CAR T cell therapy is administered.
- Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol.
- Considered by the investigator to be unable to tolerate a lymphodepleting regimen.
- Pregnancy or lactation
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Seattle Children's Hospital
Seattle, Washington, 98105
Danielle Kirkey, MD, 206-987-2000, CBDCIntake@seattlechildrens.org
Colleen Annesley, MD, 206-987-2106, CBDCIntake@seattlechildrens.org
About this study
Official title: ADVANCE-01: A Phase 1 Study of CLEC2A CAR T for Pediatric and Adult Patients With Relapsed or Refractory AML
This is a phase I, open-label, non-randomized clinical trial that will enroll pediatric and adult research participants with CLEC2A+ acute myeloid leukemia (AML) that does not go away with treatment (is refractory) or comes back after treatment (is recurrent). The goal is to evaluate the safety, feasibility, and efficacy of administering T cell products (modified immune cells) derived from the research participant's blood that have been genetically modified to express chimeric antigen receptor (CAR) targeting SC-CAR.CLEC2Av1. The main questions it seeks to answer are:
- How safe is the T cell product infusion (modified immune cells)?
- How feasible is it to manufacture the cell products? Participants will first receive treatments to prepare their body (bridging therapy and lymphodepletion). On Day 0, they receive a single infusion of CLEC2A CAR T cells, which are modified immune cells designed to target and fight cancer.
Researchers will evaluate the safety and side effects of the treatment, check how well the CAR T cells survive and multiply in the body over time (using blood, bone marrow, and cerebrospinal fluid samples), and assess how well the treatment fights the AML. Bone marrow tests are performed on Day 28 to check the response.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukaemia Recurrent, Acute Myeloid Leukaemia Refractory
- Being tested
- Biological SC-CAR.CLEC2Av1
- Keywords
- CAR T Cell, Pediatric, Adult, Acute Myeloid Leukemia Recurrent, Acute Myeloid Leukemia Refractory
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biological SC-CAR.CLEC2Av1Biological
Subjects will receive a single dose of CLEC2A CAR T cells. CD4+ and CD8+ T cell subsets will be isolated from an apheresis product obtained from the research participant. The isolated cells will be stimulated with a anti-CD3/28 activation reagent beads and transduced with a self-inactivating (SIN) lentiviral vector that directs the expression of the CLEC2A CAR.
| Study group | Role | What they receive |
|---|---|---|
| SC-CAR.CLEC2Av1 | Experimental | Autologous T cell product that has been genetically modified to express chimeric antigen receptor (CAR) targeting (SC-CAR.CLEC2Av1). Receives: Biological: Biological SC-CAR.CLEC2Av1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants experiencing Dose Limiting Toxicities (DLTs) and treatment-related adverse events as assessed by NCI CTCAE v6.0, ASTCT CRS/ICANS scales, and IEC-HS grading.
Evaluation of safety, tolerability, and full toxicity profile following a single infusion of SC-CAR.CLEC2Av1 CAR T cells. DLTs are evaluated within 28 days post-infusion to determine the Maximum Tolerated Dose (MTD) or Biologically Effective Dose (BED). Toxicities will be graded using NCI CTCAE v6.0 for general non-hematologic/hematologic toxicities, protocol-specific scales for Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and ASTCT consensus guidelines for Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS).
Measured: 28 days post-infusion
The feasibility of manufacturing SC-CAR.CLEC2Av1 CAR T cells from children and adults with relapsed or refractory CLEC2A+ AML.
Measured: 28 days
Secondary outcomes (1)
Number of participants with persistence of the SC-CAR.CLEC2Av1 CAR T-Cells
Patients will be followed for 42 days to determine if the transferred T-cells remain detectable in the blood, bone marrow, and cerebrospinal fluid.
Measured: 42 days post-infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2026
- Study startNov 2026 (estimated)
- Primary completionFeb 2030 (estimated)
- Study completionFeb 2043 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Seattle Children's HospitalThe sponsor's other registered studies.
- 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Seattle Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Colleen Annesley, Seattle Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07905027
- Sponsor's protocol ID
- ADVANCE-01
- Acronym
- ADVANCE-01
- Status last verified
- Sep 2026
- First submitted
- 22 Sep 2026
- Last updated
- 2 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ADVANCE-01: A Phase 1 Study of CLEC2A CAR T for Pediatric and Adult Patients With Relapsed or Refractory AML
- ClinicalTrials.gov ID
- NCT07905027
- Acronym / sponsor ID
- ADVANCE-01 / ADVANCE-01
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukaemia Recurrent, Acute Myeloid Leukaemia Refractory
- Keywords
- CAR T Cell, Pediatric, Adult, Acute Myeloid Leukemia Recurrent, Acute Myeloid Leukemia Refractory
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Seattle Children's HospitalUniversity, hospital or other
- Study start
- Nov 2026 (estimated)
- Primary completion
- Feb 2030 (estimated)
- Study completion
- Feb 2043 (estimated)
- First posted
- 29 Oct 2026 (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.
This is a phase I, open-label, non-randomized clinical trial that will enroll pediatric and adult research participants with CLEC2A+ acute myeloid leukemia (AML) that does not go away with treatment (is refractory) or comes back after treatment (is recurrent). The goal is to evaluate the safety, feasibility, and efficacy of administering T cell products (modified immune cells) derived from the research participant's blood that have been genetically modified to express chimeric antigen receptor (CAR) targeting SC-CAR.CLEC2Av1. The main questions it seeks to answer are:
- How safe is the T cell product infusion (modified immune cells)?
- How feasible is it to manufacture the cell products? Participants will first receive treatments to prepare their body (bridging therapy and lymphodepletion). On Day 0, they receive a single infusion of CLEC2A CAR T cells, which are modified immune cells designed to target and fight cancer.
Researchers will evaluate the safety and side effects of the treatment, check how well the CAR T cells survive and multiply in the body over time (using blood, bone marrow, and cerebrospinal fluid samples), and assess how well the treatment fights the AML. Bone marrow tests are performed on Day 28 to check the response.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SC-CAR.CLEC2Av1 | Experimental | Biological: Biological SC-CAR.CLEC2Av1Autologous T cell product that has been genetically modified to express chimeric antigen receptor (CAR) targeting (SC-CAR.CLEC2Av1). |
Who may qualify
- Ages
- Up to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (27 listed)
- Subject age ≤ 70 years
- Weight ≥ 7 kilograms
- AML that expresses CLEC2A by flow cytometry as assessed by Hematologics, Inc. Laboratory and meets one of the below definitions:
- Able to tolerate apheresis
- Life expectancy ≥ 8 weeks
Key exclusion criteria (15 listed)
- Active malignancy other than acute myeloid leukemia.
- History of symptomatic non-AML CNS disease or ongoing symptomatic CNS disease requiring medical intervention, including paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injury, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder (subjects with non-febrile seizure disorder controlled on anti-epileptic medication and without seizure activity within 1 month are eligible).
- CNS AML involvement that is symptomatic and in the opinion of the investigator, cannot be controlled during the interval between enrollment and T cell infusion.
- If history of allogeneic stem cell transplant: active GVHD or receiving immunosuppressive therapy for treatment or prevention of GVHD within 4 weeks prior to enrollment.
- If history of allogeneic stem cell transplant and patient has received donor lymphocyte infusion (DLI) the subject is <8 weeks from DLI infusion.
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 |
|---|---|---|
| Biological SC-CAR.CLEC2Av1Subjects will receive a single dose of CLEC2A CAR T cells. CD4+ and CD8+ T cell subsets will be isolated from an apheresis product obtained from the research participant. The isolated cells will be stimulated with a anti-CD3/28 activation reagent beads and transduced with a self-inactivating (SIN) lentiviral vector that directs the expression of the CLEC2A CAR. | Biological | SC-CAR.CLEC2Av1 |
Primary outcome
Number of participants experiencing Dose Limiting Toxicities (DLTs) and treatment-related adverse events as assessed by NCI CTCAE v6.0, ASTCT CRS/ICANS scales, and IEC-HS grading.
Time frame: 28 days post-infusion
Evaluation of safety, tolerability, and full toxicity profile following a single infusion of SC-CAR.CLEC2Av1 CAR T cells. DLTs are evaluated within 28 days post-infusion to determine the Maximum Tolerated Dose (MTD) or Biologically Effective Dose (BED). Toxicities will be graded using NCI CTCAE v6.0 for general non-hematologic/hematologic toxicities, protocol-specific scales for Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and ASTCT consensus guidelines for Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS).
The feasibility of manufacturing SC-CAR.CLEC2Av1 CAR T cells from children and adults with relapsed or refractory CLEC2A+ AML.
Time frame: 28 days
1 secondary outcome
Number of participants with persistence of the SC-CAR.CLEC2Av1 CAR T-Cells
Time frame: 42 days post-infusion.
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 2 Nov 2026.
- Individual participant dataSharing planned: Undecided
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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.
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 Biological SC-CAR.CLEC2Av1 in people with Acute Myeloid Leukemia and Acute Myeloid Leukaemia Recurrent, run by Seattle Children's Hospital 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 27 inclusion and 15 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: opening soon, as of the sponsor's last update on 2 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 Danielle Kirkey, MD (206-987-2000, CBDCIntake@seattlechildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants experiencing Dose Limiting Toxicities (DLTs) and treatment-related adverse events as assessed by NCI CTCAE v6.0, ASTCT CRS/ICANS scales, and IEC-HS grading. (time frame: 28 days post-infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Nov 2026.