Adult Acute Myeloid Leukemia in RemissionPhase 1NCT02159495

Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

A Phase 1 study testing cyclophosphamide, Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes and laboratory biomarker analysis in people with Adult Acute Myeloid Leukemia in Remission and Acute Biphenotypic Leukemia, run by City of Hope Medical Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

Who is it for?: Ages 12 years and older
Conditions the study is for: Adult Acute Myeloid Leukemia in Remission, Acute Biphenotypic Leukemia, Early Relapse of Acute Myeloid Leukemia
What is being tested?: cyclophosphamide, Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes and laboratory biomarker analysis
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 31 participants
Where: 1 siteUnited States
Study dates: 15 Dec 2015to 14 Jul 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 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

ARM 1 - AML: Research patients enrolled are those patients with relapsed or refractory CD123+ AML de novo, or secondary OR participants who are at high risk for disease recurrence NOTE: CD123+ biphenotypic acute leukemia or CD123+ acute lymphoblastic leukemia (ALL) may also be considered but only after discussion with the study principal investigator (PI)
Relapsed AML is defined as patients that had a first complete remission (CR) before developing recurrent disease (increased bone marrow blasts)
Refractory AML is defined as patients that have not achieved a first CR after 2 cycles of induction chemotherapy; for patients with AML evolving from myelodysplastic syndrome, they should have completed at least one cycle of induction chemotherapy
ARM 2 - BPDCN: Research participants with a diagnosis of BPDCN, according to World Health Organization (WHO) classification by hematopathology, who underwent at least 1 line of systemic therapy for BPDCN and who have persistent or recurrent disease in at least one of the following are eligible: peripheral blood, bone marrow, lymph nodes, spleen, cutaneous lesions or other sites OR participant who are at high risk for disease recurrence
FOR BOTH STUDY ARMS: Research participants must have bone marrow and/or peripheral blood samples available for confirmation of diagnosis of AML or BPDCN; CD123 positivity must be confirmed by either flow cytometry or immunohistochemistry within 90 days of study entry; cytogenetics, flow cytometry, and molecular studies (such as FMS-like tyrosine kinase-3 [FLT-3] status) will be obtained as per standard practice; however, for research participants who are at a high risk of recurrence, they must have historical bone marrow and/or peripheral blood samples available for confirmation of diagnosis of AML or BPDCN; CD123 positivity must be confirmed by either flow cytometry or immunohistochemistry prior to start of lymphodepletion
Karnofsky performance status score >= 70

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A life expectancy >= 16 weeks at time of enrollment
Pediatric research participants must weigh > 50 kg
Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for six months following duration of study participation; should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately
Calculated creatinine clearance (absolute value) of >= 50 mL/minute or creatinine < 2.0 mg/dl or < 2 times upper limit of normal for the research participant's age group
Serum bilirubin =< 3.0 mg/dL
Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =< 5 times the institutional upper limits of normal
Ejection fraction measured by echocardiogram (ECHO) or multi gated acquisition scan (MUGA) >= 50%
ONLY research participants experiencing hypoxia with oxygen saturation less than 92% are required to have diffusion capacity of carbon monoxide (DLCO) or forced expiratory volume in one second (FEV1) > 45% predicted
Research participants' last dose of prior chemotherapy or radiation must be >= 2 weeks before leukapheresis
Note: the above criterion is not applicable if the research participant's donor is undergoing leukapheresis
If a research participant has undergone prior allogeneic stem cell transplant, he/she must be off all immunosuppressants for graft versus host disease (GVHD) for at least 2 weeks before undergoing leukapheresis
Note: the above criterion is not applicable if the research participant's donor is undergoing leukapheresis
Negative serum or urine pregnancy test
All research participants must have the ability to understand and willingness to sign a written informed consent or age appropriate assent for pediatric patients
Note: For research participants who do not speak English, a short form consent may be used with a City of Hope (COH) certified interpreter/translator to proceed with screening and leukapheresis, while the request for a translated full consent is processed; however, the research participant is allowed to proceed with lymphodepletion and T cell infusion only after the translated full consent form is signed
ELIGIBILITY TO PROCEED WITH PERIPHERAL BLOOD MONONUCLEAR CELL (PMBC) COLLECTION:
If research participant is undergoing leukapheresis:
He/she has acceptable venous access as assessed by Donor Apheresis Center or if venous access was not acceptable, a Hickman Catheter or temporary line was placed prior to scheduled leukapheresis
He/she has undergone prior alloSCT, they must be at least 2 weeks from having received the last dose of immunosuppressant medications to undergo PBMC collection for T cell manufacturing
His/her last dose of prior chemotherapy, immunotherapy or radiation is at least 2 weeks out from PBMC collection
ELIGIBILITY TO UNDERGO LYMPHODEPLETION Note: evaluations should be performed no more than 7 days prior to lymphodepletion
Research participant with central nervous system (CNS) leukemic involvement that is refractory to intrathecal chemotherapy and/or cranio-spinal radiation but effectively treated to completion remission (< 5 white blood cell[WBC]/mm^3 and no blast in cerebrospinal fluid [CSF]) is eligible to proceed with lymphodepletion
Research participants must have a donor or stem cells source identified for allogeneic transplantation, either related (7/8 or 8/8 allele matched or haploidentical), unrelated 7/8 or 8/8 allele match) donor, or cord blood stem cell source (at lease 4/6 matched)
Research participants with a response less than a CR or complete response with incomplete hematopoietic recovery (CRi) or detectable minimal residual disease (MRD) positive disease
Research participant has a released cryopreserved T cell product for CAR T cell infusion on approximately day 0
Research participant must be at least 2 weeks out from having received the last dose of investigational agent
Karnofsky performance status (KPS) >= 70
Documented measurable or evaluable disease
Non hematological toxicity related to prior therapy must either have returned to =< grade 2, baseline, or deemed irreversible
Research participants of reproductive potential must agree to use and utilize and adequate method of contraception throughout treatment and for at least 8 weeks after T cell infusion
If a research participant has undergone prior allogeneic stem cell transplant, he/she must be off all immunosuppressants for GVHD for at least 7 days before beginning lymphodepletion
Pulmonary: not requiring supplemental oxygen or mechanical ventilation, oxygen saturation 90% or higher on room air
Cardiovascular: not requiring pressor support, no symptomatic cardiac arrhythmias, no acute coronary syndrome, or uncontrolled hypertension
Renal Function: calculated creatinine clearance (absolute value) of >= 50 mL/minute or creatinine < 2.0 mg/dl or < 2 times upper limit of normal for the research participant's age group
Liver Function: adequate liver function defined as total bilirubin =< 3.0 mg/dl
ALT and AST =< 5 times the institutional upper limits of normal
Neurological: research participant without clinically significant encephalopathy/new focal deficits
Infectious diseases: no clinical evidence of uncontrolled active infectious process
ELIGIBILITY CRITERIA AT TIME OF INFUSION OF GENETICALLY MODIFIED T CELLS
Research participants has undergone lymphodepletion
Pulmonary: not requiring supplemental oxygen or mechanical ventilation, oxygen saturation 90% or higher on room air
Cardiovascular: not requiring pressor support, no symptomatic cardiac arrhythmias, no acute coronary syndrome, or uncontrolled hypertension
Renal Function: calculated creatinine clearance (absolute value) of >= 50 mL/minute or creatinine < 2.0 mg/dl or < 2 times upper limit of normal for the research participant's age group
Liver Function: adequate liver function defined as total bilirubin =< 3.0 mg/dl
ALT and AST =< 5 times the institutional upper limits of normal
Neurological: research participant without clinically significant encephalopathy/new focal deficits
Infectious diseases: no clinical evidence of uncontrolled active infectious process
Research participant must be off all anti-leukemic drugs, with the exception of the lymphodepleting regimens, at least 7 days prior to CAR T cell infusion
ELIGIBILITY CRITERIA TO UNDERGO OPTIONAL T CELL ABLATION
Research participant has >= 1% CAR T cells in the peripheral blood
Pulmonary: not requiring supplemental oxygen or mechanical ventilation, oxygen saturation 90% or higher on room air
Cardiovascular: not requiring pressor support, no symptomatic cardiac arrhythmias, no acute coronary syndrome, or uncontrolled hypertension
Renal Function: serum creatinine did NOT increase by more than 2.5 fold from baseline (at time of screening)
Liver Function: adequate liver function defined as total bilirubin =< 3.0 mg/dl
AST =< 5 x ULN, ALT =< 5 x ULN
Neurological: research participant without clinically significant encephalopathy/new focal deficits
Infectious diseases: no clinical evidence of uncontrolled active infectious process
ALLOGENEIC DONOR CRITERIA FOR APHERESIS DONATION:
Related donor selection will be conducted in accordance with City of Hope's Department of Hematology \& Hematopoietic Cell Transplantation criteria and, in the case of unrelated donor from a transplant center, will comply with the National Marrow Donor Program's (NMDP) donor selection standards; when a potentially eligible recipient of an unrelated donor product from an NMDP Center is identified, the recipient will complete an NMDP search transfer request to allow City of Hope (COH) NMDP staff to contact the NMDP Coordinating Center, who in turn, will contact the donor's prior Donor Center; the search will follow the NMDP Policy for subsequent donation requests; any form deemed appropriate and necessary by the NMDP, including the Subsequent Donation Request Form, Therapeutic T Cell Collection Prescription and Therapeutic Stem Cell Collection Prescription, will be submitted as required
In the case of a related donor: The identified donor must be the original donor whose stem cells were used for the research participant's allogeneic stem cell transplantation (alloSCT)
For both related and unrelated donors: The donor's hepatitis B surface antigen must be negative and the hepatitis C antibody must be nonreactive; in the case of a positive hepatitis C antibody result, the hepatitis C virus (HCV) viral polymerase chain reaction (PCR) will have to be performed and the results should be negative

Who cannot join

Research participants with uncontrolled intercurrent illness including, but not limited to ongoing or active or poorly controlled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, poorly controlled pulmonary disease or psychiatric illness/social situations that would limit compliance with study requirements; such social situations include but are not limited to lack of reliable means of transportation for follow up, inability to make time for required clinic visits due to work or family needs, or lack of reliable ways of communication with the study team in the event that the participant is seriously ill
Research participants who have tested human immunodeficiency virus (HIV) positive, or have active hepatitis B or C infection based on testing performed within 4 weeks of enrollment
Research participants with presence of other active malignancy. However, research participants with history of prior malignancy treated with curative intent and in complete remission are eligible
Pregnant and lactating women are excluded from this study
Study-Specific Exclusion
Failure of research participant to understand the basic elements of the protocol and/or the risks/benefits of participating in this phase I study

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History of allergic reactions attributed to compounds of similar chemical or biological composition to cetuximab
Dependence on corticosteroids:
If the participant is undergoing leukapheresis: physiological replacement doses of steroids are allowed - prednisone no more than 7.5 mg, hydrocortisone less than 12 mg/m^2/day
However, all participants must be able to reduce steroid requirement to no more than physiological replacement doses prior to start of lymphodepletion
Active autoimmune disease requiring systemic immunosuppressive therapy
Research participants will be excluded, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study

Trial locations and contacts

1 site in 1 country.

United States1 site

City of Hope Medical Center
Duarte, California, 91010

Other trials in United States

About this study

Official title: Phase I Study of Cellular Immunotherapy Using T Cells Lentivirally Transduced to Express a CD123-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR for Patients With CD123+ Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

This phase I trial studies the side effects and the best dose of genetically modified T-cells after lymphodepleting chemotherapy in treating patients with acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm that has returned after a period of improvement or has not responded to previous treatment. An immune cell is a type of blood cell that can recognize and kill abnormal cells in the body. The immune cell product will be made from patient or patient's donor (related or unrelated) blood cells. The immune cells are changed by inserting additional pieces of deoxyribonucleic acid (DNA) (genetic material) into the cell to make it recognize and kill cancer cells. Placing a modified gene into white blood cells may help the body build an immune response to kill cancer cells.

Read the detailed description

PRIMARY OBJECTIVES: I. To examine the anti-tumor activity and safety of administering ex vivo expanded T cells that are genetically modified using a self-inactivating (SIN) lentiviral vector to express a co-stimulatory CD123-specific chimeric antigen receptor (CAR) as well as a truncated EGFR (CD123CAR-CD28-CD3zeta-EGFRt+ T cells [CD123+ CAR T cells]) following lymphodepletion for patients with CD123+ relapsed or refractory acute myeloid leukemia (AML) (arm 1), or CD123+ persistent or recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) (arm 2). II. To determine the recommended Phase II dose (RP2D) for both arms (AML and BPDCN). SECONDARY OBJECTIVES: I. To assess activity in the form of CD123+ CAR T cell persistence, 6 month progression free survival (PFS 6mo) rate, and 1 year overall survival (OS) rate, and describe the immunogenicity of CD123R(EQ)28zeta/EGFRt+ T cells. TERTIARY OBJECTIVES: I. To assess impact on hematopoiesis, change from baseline in numbers of CD123+ blood cells, CD123 expression on malignant cells and hematopoietic cells, and the clinical efficacy of EGFRt mediated CAR T cell ablation.

OUTLINE: This is a dose-escalation study of autologous or allogeneic (related or unrelated donor) CD123+ CAR T cells. Patients undergo a lymphodepleting regimen 3-10 days prior to CD123+ CAR T cell infusion as determined by the principal investigator and the protocol team. Patients receive either cyclophosphamide intravenously (IV) on days -4 and/or -3; fludarabine phosphate and cyclophosphamide IV on days -5 to -3; fludarabine phosphate IV on days -5 to -3 and cyclophosphamide IV on days -4 and/or -3. Patients receive autologous or allogeneic CD123+ CAR T cells IV over 15 minutes on day 0. Patients with evidence of disease at > 28 days, continuing expression of the CD123 antigen, and not having experienced a dose-limiting toxicity (DLT) may receive a second infusion of CD123+ CAR T cells after 28 days. After completion of study treatment, patients are followed up at 24 hours, then every 2 days for up to 14 days, every week for 1 month, every month for 1 year and then yearly for 15 years.

Conditions: Adult Acute Myeloid Leukemia in Remission, Acute Biphenotypic Leukemia, Early Relapse of Acute Myeloid Leukemia, Late Relapse of Acute Myeloid Leukemia, Recurrent Adult Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia
Being tested: cyclophosphamide, Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes, laboratory biomarker analysis, Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytes, Fludarabine Phosphate
Medical subject headings: Leukemia, Biphenotypic, Acute, Leukemia, Myeloid, Acute, Blastic Plasmacytoid Dendritic Cell Neoplasm, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Neoplasm, Residual

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

cyclophosphamideDrug
Also called CPM, CTX, Cytoxan, Endoxan
Given IV
Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T LymphocytesBiological
Also called CD123R(EQ)28zeta/EGFRt+ T cells, Anti CD123-CAR/CD28-costimulatory Lentiviral Vector-transduced Autologous T Lymphocytes
Given IV
laboratory biomarker analysisOther
Correlative studies
Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytesBiological
Also called Allogeneic CD123R(EQ)28zeta/EGFRt+ T Cells
Given IV
Fludarabine PhosphateDrug
Also called 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara
Given IV

Study groups in NCT02159495
Study group	Role	What they receive
Treatment (lymphodepletion, T-cell immunotherapy)	Experimental	Patients undergo a lymphodepleting regimen 3-10 days prior to CD123+ CAR T cell infusion as determined by the principal investigator and the protocol team. Patients receive either cyclophosphamide IV on days -4 and/or -3; fludarabine phosphate and cyclophosphamide IV on days -5 to -3; fludarabine phosphate IV on days -5 to -3 and cyclophosphamide IV on days -4 and/or -3. Patients receive autologous or allogeneic CD123+ CAR Tcells IV over 15 minutes on day 0. Patients with evidence of disease at > 28 days, continuing expression of the CD123 antigen, and not having experienced a DLT may receive a second infusion of CD123+ CAR T cells after 28 days. Receives: Drug: cyclophosphamide; Biological: Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes; Other: laboratory biomarker analysis; Biological: Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytes; Drug: Fludarabine Phosphate

What is being measured

The primary outcome is the main question the study is designed to answer.

Dose-limiting toxicity (DLT) defined as any grade 3 or higher toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Tables will summarize all toxicities and side effects by attribution of treatment, dose, time-post treatment, organ and severity. Analysis will be done separately for each disease arm.
Measured: 28 days
Incidence of adverse events as assessed by NCI CTCAE version 4.0
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Tables will summarize all toxicities and side effects by attribution of treatment, dose, time-post treatment, organ and severity. Analysis will be done separately for each disease arm.
Measured: Up to 15 years
Disease response (CR or CRi)
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Analysis will be done separately for each disease arm.
Measured: Up to 15 year post-treatment

Secondary outcomes (5)

Engraftment of transferred CD123+ CAR T cells
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Analysis will be done separately for each disease arm.
Measured: Day 28
CAR123-specific antibody level
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Analysis will be done separately for each disease arm.
Measured: Up to 15 years
Duration of response
Will provide descriptive statistics. Analysis will be done separately for each disease arm.
Measured: Up to 15 years
Progression Free Survival (PFS)
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated for PFS 6mo. Analysis will be done separately for each disease arm.
Measured: Up to 15 years
Survival
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated for 1 year OS. Analysis will be done separately for each disease arm.
Measured: Up to 15 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Dec 201514 Jul 2027

First posted10 Jun 2014 (estimated)
Study start15 Dec 2015
Primary completion14 Jul 2027 (estimated)
Study completion14 Jul 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Adult Acute Myeloid Leukemia in RemissionStudies of the same condition, open and under way.
More trials run by City of Hope Medical CenterThe 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: Single group
Masking: None (open label)
Enrolment: 31 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02159495
Sponsor's protocol ID: 13272
Other IDs: NCI-2014-01147, 13272
Status last verified: Aug 2026
First submitted: 6 Jun 2014
Last updated: 26 Aug 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Budde LE, Del Real MM, Song J, Stiller T, Wang Y, Aribi A, Sandhu K, Nakamura R, Marcucci EC, Wu JJ, Ng K, Clark MC, Wood B, Wagner JR, Paul J, Brown CE, Stein A, Marcucci G, Blanchard MS, Palmer J, Forman SJ. A phase 1 trial of CD123-directed chimeric antigen receptor T-cell therapy in adults with relapsed/refractory acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm. J Hematol Oncol. 2026 Aug 7;19(1):68. doi: 10.1186/s13045-026-01833-3.
PubMed 42681647

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase I Study of Cellular Immunotherapy Using T Cells Lentivirally Transduced to Express a CD123-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR for Patients With CD123+ Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm
ClinicalTrials.gov ID: NCT02159495
Acronym / sponsor ID: 13272
Conditions: Adult Acute Myeloid Leukemia in Remission, Acute Biphenotypic Leukemia, Early Relapse of Acute Myeloid Leukemia, Late Relapse of Acute Myeloid Leukemia, Recurrent Adult Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia
Phase: Phase 1
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 31 participants (actual)
Lead sponsor: City of Hope Medical CenterUniversity, hospital or other
Collaborators: National Cancer Institute (NCI), Mustang Bio, Inc.
Study start: 15 Dec 2015 (actual)
Primary completion: 14 Jul 2027 (estimated)
Study completion: 14 Jul 2027 (estimated)
First posted: 10 Jun 2014 (estimated)
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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment (lymphodepletion, T-cell immunotherapy)	Experimental	Drug: cyclophosphamide, Biological: Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes, Other: laboratory biomarker analysis, Biological: Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytes, Drug: Fludarabine PhosphatePatients undergo a lymphodepleting regimen 3-10 days prior to CD123+ CAR T cell infusion as determined by the principal investigator and the protocol team. Patients receive either cyclophosphamide IV on days -4 and/or -3; fludarabine phosphate and cyclophosphamide IV on days -5 to -3; fludarabine phosphate IV on days -5 to -3 and cyclophosphamide IV on days -4 and/or -3. Patients receive autologous or allogeneic CD123+ CAR Tcells IV over 15 minutes on day 0. Patients with evidence of disease at > 28 days, continuing expression of the CD123 antigen, and not having experienced a DLT may receive a second infusion of CD123+ CAR T cells after 28 days.

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (67 listed)

ARM 1 - AML: Research patients enrolled are those patients with relapsed or refractory CD123+ AML de novo, or secondary OR participants who are at high risk for disease recurrence NOTE: CD123+ biphenotypic acute leukemia or CD123+ acute lymphoblastic leukemia (ALL) may also be considered but only after discussion with the study principal investigator (PI)
ARM 2 - BPDCN: Research participants with a diagnosis of BPDCN, according to World Health Organization (WHO) classification by hematopathology, who underwent at least 1 line of systemic therapy for BPDCN and who have persistent or recurrent disease in at least one of the following are eligible: peripheral blood, bone marrow, lymph nodes, spleen, cutaneous lesions or other sites OR participant who are at high risk for disease recurrence
FOR BOTH STUDY ARMS: Research participants must have bone marrow and/or peripheral blood samples available for confirmation of diagnosis of AML or BPDCN; CD123 positivity must be confirmed by either flow cytometry or immunohistochemistry within 90 days of study entry; cytogenetics, flow cytometry, and molecular studies (such as FMS-like tyrosine kinase-3 [FLT-3] status) will be obtained as per standard practice; however, for research participants who are at a high risk of recurrence, they must have historical bone marrow and/or peripheral blood samples available for confirmation of diagnosis of AML or BPDCN; CD123 positivity must be confirmed by either flow cytometry or immunohistochemistry prior to start of lymphodepletion
Karnofsky performance status score >= 70
A life expectancy >= 16 weeks at time of enrollment

Key exclusion criteria (12 listed)

Research participants with uncontrolled intercurrent illness including, but not limited to ongoing or active or poorly controlled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, poorly controlled pulmonary disease or psychiatric illness/social situations that would limit compliance with study requirements; such social situations include but are not limited to lack of reliable means of transportation for follow up, inability to make time for required clinic visits due to work or family needs, or lack of reliable ways of communication with the study team in the event that the participant is seriously ill
Research participants who have tested human immunodeficiency virus (HIV) positive, or have active hepatitis B or C infection based on testing performed within 4 weeks of enrollment
Research participants with presence of other active malignancy. However, research participants with history of prior malignancy treated with curative intent and in complete remission are eligible
Pregnant and lactating women are excluded from this study
Study-Specific Exclusion

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
cyclophosphamideAlso: CPM, CTX, Cytoxan, EndoxanGiven IV	Drug	Treatment (lymphodepletion, T-cell immunotherapy)
Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T LymphocytesAlso: CD123R(EQ)28zeta/EGFRt+ T cells, Anti CD123-CAR/CD28-costimulatory Lentiviral Vector-transduced Autologous T LymphocytesGiven IV	Biological	Treatment (lymphodepletion, T-cell immunotherapy)
laboratory biomarker analysisCorrelative studies	Other	Treatment (lymphodepletion, T-cell immunotherapy)
Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytesAlso: Allogeneic CD123R(EQ)28zeta/EGFRt+ T CellsGiven IV	Biological	Treatment (lymphodepletion, T-cell immunotherapy)
Fludarabine PhosphateAlso: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, FludaraGiven IV	Drug	Treatment (lymphodepletion, T-cell immunotherapy)

Primary outcome

Dose-limiting toxicity (DLT) defined as any grade 3 or higher toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Time frame: 28 days
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Tables will summarize all toxicities and side effects by attribution of treatment, dose, time-post treatment, organ and severity. Analysis will be done separately for each disease arm.
Incidence of adverse events as assessed by NCI CTCAE version 4.0
Time frame: Up to 15 years
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Tables will summarize all toxicities and side effects by attribution of treatment, dose, time-post treatment, organ and severity. Analysis will be done separately for each disease arm.
Disease response (CR or CRi)
Time frame: Up to 15 year post-treatment
Rates and associated 95% Clopper and Pearson binomial confidence limits will be estimated. Analysis will be done separately for each disease arm.

5 secondary outcomes

Engraftment of transferred CD123+ CAR T cells
Time frame: Day 28
CAR123-specific antibody level
Time frame: Up to 15 years
Duration of response
Time frame: Up to 15 years
Progression Free Survival (PFS)
Time frame: Up to 15 years
Survival
Time frame: Up to 15 years

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 26 Aug 2026.
Derived from this study: PubMed 42681647Budde LE, Del Real MM, Song J, Stiller T, Wang Y, Aribi A, Sandhu K, Nakamura R, Marcucci EC, Wu JJ, Ng K, Clark MC, Wood B, Wagner JR, Paul J, Brown CE, Stein A, Marcucci G, Blanchard MS, Palmer J, Forman SJ. A phase 1 trial of CD123-directed chimeric antigen receptor T-cell therapy in adults with relapsed/refractory acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm. J Hematol Oncol. 2026 Aug 7;19(1):68. doi: 10.1186/s13045-026-01833-3.

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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. This study is not recruiting at the moment.

Lihua E. Budde principal investigator

City of Hope Medical Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 67 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: under way, not looking for participants, as of the sponsor's last update on 26 Aug 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Dose-limiting toxicity (DLT) defined as any grade 3 or higher toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (time frame: 28 days). The study lists 3 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 NCT02159495, which also lists 1 linked publication.

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 26 Aug 2026; AzTrials last refreshed its copy on 26 Oct 2026.