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CD19+ Acute LeukemiaPhase 1/2NCT02028455

A Pediatric and Young Adult Trial of Genetically Modified T Cells Directed Against CD19 for Relapsed/Refractory CD19+ Leukemia

A Phase 1/2 study testing Patient Derived CD19 specific CAR T cells also expressing an EGFRt in people with CD19+ Acute Leukemia, run by Seattle Children's Hospital at 3 sites in United States.

Active, not recruiting

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

Official record
Search all trials

Trial summary

Patients with relapsed or refractory leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use T cells obtained directly from the patient, which can be genetically modified to express a chimeric antigen receptor (CAR). Read more about this study

Who is it for?
Ages 1 year to 26 years
Conditions the study is for
CD19+ Acute Leukemia
What is being tested?
Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Could I receive a placebo?
No, everyone receives an active treatment
How many people
167 participants
Where
3 sitesUnited States
Study dates
11 Feb 2014to Jul 2036
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 1 year to 26 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients must be ≥12 months of age and <27 years of age at the time of study enrollment.
  • Must be ≥10kg
  • Confirmed CD19+ leukemia recurrence defined as ≥0.01% disease in the marrow or isolated extramedullary disease following allogeneic HCT. [N.B. Study closed to enrollment of leukemia subjects]
  • OR
  • No prior history of allogeneic HCT (one of the following)
  • 2nd or greater relapse, with or without extramedullary disease (isolated extramedullary disease is eligible)
Show all 35Show fewer
  • 1st marrow relapse at end of 1st month of re-induction with marrow having ≥0.01% blast disease, with or without extramedullary disease
  • Primary Refractory as defined as having M2 or M3 marrow after induction
  • Subject has indication for HCT but has been deemed ineligible
  • OR
  • CD19+ Non-Hodgkin Lymphoma (NHL) refractory or relapsed with no known curative therapies available [N.B. Study remains open to enrollment of lymphoma subjects]
  • Patients with CNS involvement are eligible provided that they are asymptomatic and in the opinion of the study PI have a reasonable expectation that disease burden can be controlled in the interval between enrollment and T cell infusion. Patients that have a significant neurologic deterioration will be not be eligible for T cell infusion until alternate therapies result in neurological stabilization.
  • Patients must have a Lansky performance status score of ≥50 or a Karnofsky score of ≥ 50 for patients ≥16 years of age.
  • Life Expectancy of >8 weeks
  • Patients must be free from active GVHD and off immunosuppressive GVHD therapy for 4 weeks prior to enrollment.
  • Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy
  • It must be at least 7 days since last chemotherapy was administered (this does not include intrathecal chemotherapy or maintenance chemotherapy)
  • No systemic corticosteroids (unless physiologic replacement dosing) within 7 days of enrollment.
  • No prior genetically modified cell therapy that is still detectable or virotherapy allowed.
  • Normal serum creatinine based on age/gender
  • Total bilirubin </3x ULN OR conjugated bilirubin </2mg/dl
  • ALT </5X ULN
  • SF of >28% by ECHO or EF >50% by MUGA
  • ALC of >/= 100 cells/ul
  • Pulse ox >/= 90% on room air
  • Patient must have documented negative HIV antigen and antibody, Hepatitis B surface antigen, and Hepatitis C antibody within 3 months prior to enrollment. For patient with positive Hepatitis C Ab, negative PCR testing must be documented in order to be eligible.
  • Patients must NOT have active clinically significant CNS dysfunction (including but not limited to such as uncontrolled seizure disorder, paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injuries, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder)
  • Must agree to highly effective contraception during and for 12 months after T cell infusion.
  • Patients must be able to tolerate apheresis procedure, including placement of temporary apheresis line if required.
  • Patients must NOT have an active malignancy other than CD19+ leukemia.
  • Patients must NOT have an active severe infection defined as:
  • A positive blood culture within 48 hours of study enrollment
  • A fever above 38.2 C AND clinical signs of infection within 48 hours of study enrollment
  • Patients must NOT have any concurrent medical condition that, in the opinion of the PI or designee, would prevent the patient from undergoing protocol-based therapy. Patients with a primary immunodeficiency/ bone marrow failure syndrome are excluded from this trial.
  • Research participant or parent/legal guardian must agree to participate in long-term follow-up for up to 15 years, if they are enrolled in the study and receive T-cell infusion.

Who cannot join

None listed.

Trial locations and contacts

3 sites in 1 country.

United States3 sites
  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • Children's Hospital Oakland

    Oakland, California, 94609

  • Seattle Children's Hospital

    Seattle, Washington, 98105

Other trials in United States

About this study

Official title: Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-02: A Phase 1/2 Feasibility and Safety Study of CD19-CAR T Cell Immunotherapy for CD19+ Leukemia

Patients with relapsed or refractory leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use T cells obtained directly from the patient, which can be genetically modified to express a chimeric antigen receptor (CAR). The CAR enables the T cell to recognize and kill the leukemic cell through the recognition of CD19, a protein expressed of the surface of the leukemic cell in patients with CD19+ leukemia. This is a phase 1/2 study designed to determine the maximum tolerated dose of the CAR+ T cells as well as to determine the efficacy. The phase 1 cohort is restricted to those patients who have already had an allogeneic hematopoietic cell transplant (HCT). The phase 2 is open to all patients regardless of having a history of HCT.

Read the detailed description

Upon meeting the eligibility requirements and enrolling on study, subjects will undergo apheresis to obtain the T cells for the generation of the CD19 CAR+ T cells. In patients with a prior history of allogeneic HCT, the T cells obtained are of donor origin. The T cells are isolated from the apheresis product, the CD4 and CD8 T cells are then selected and grown separately, transduced with a lentivirus to express the CD19 CAR as well as a truncated EGFR that has no signaling capacity (noted EGFRt) and expanded in culture over a three week period. During the process of cell generation, subjects will continue to be cared for by their primary oncologist and may undergo additional treatment directed at the leukemia during this time.

After the CAR+ T cells have been generated, the subject undergoes a disease assessment and determination if lymphodepletion is necessary. A variety of lymphodepletion strategies are acceptable and determined on a case by case basis. At least 48 hours after the completion of lymphodepletion, the subject will receive and infusion of CAR+ T cells at an approximate 1:1 ratio of CD4 to CD8 CAR+ T cells.

Following treatment with the CAR+ T cells, subjects will be followed intensely for 2 months with serial blood testing and re-evaluation of disease status with bone marrow aspirates. After 2 months, the subjects clinical care will be resumed by their primary oncologist, and it is possible that they would receive additional chemotherapy or HCT.

Some subjects will receive cetuximab for ablation of the genetically modified T cells. Criteria to receive cetuximab include acute toxicities that are life threatening, as well as an ongoing remission with continued B cell aplasia.

Upon completion of the study, subjects will be followed bi-annually for 5 years, and then annually for 10 additional years with either a medical history, physical exam and blood tests or a phone call/questionnaire. This follow up will help to determine if the subject develops any long-term health problems related to the CAR+ T cells including a new cancer.

Being tested
Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Keywords
pediatric, young adult, acute lymphoblastic leukemia, CD19, leukemia, Chimeric Antigen Receptor, T cell
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia

What participants receive

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

  • Patient Derived CD19 specific CAR T cells also expressing an EGFRtBiological

    Defined Composition CD4 and CD8 T cells Lentivirally Transduced to Express a Second Generation 4-1BB:zeta CD19 CAR and EGFRt

Study groups in NCT02028455
Study groupRoleWhat they receive
Phase 1 - Cohort 1AExperimentalThis phase 1 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 5x10^5 CAR T cells/kg
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Phase 1 - Cohort 1BExperimentalThis phase 1 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 1x10^6 CAR T cells/kg
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Phase 1 - Cohort 1CExperimentalThis phase 1 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 5x10^6 CAR T cells/kg
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Phase 1 - Cohort 1DExperimentalThis phase 1 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 1x10^7 CAR T cells/kg
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Phase 1 - Cohort 1F1ExperimentalThis phase 1 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 5x10^5 CAR T cells/kg following prescribed lymph-depletion with fludarabine and cyclophosphamide
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Phase 1 - Cohort 1F2ExperimentalThis phase 1 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 1x10^6 CAR T cells/kg following prescribed lymph-depletion with fludarabine and cyclophosphamide
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt
Phase 2ExperimentalThe phase 2 cohort will receive Patient Derived CD19 specific CAR T cells at a dose of 1 x 10^6 CAR T cells/kg following lymphodepletion if indicated.
Receives: Biological: Patient Derived CD19 specific CAR T cells also expressing an EGFRt

What is being measured

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

  1. Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity Definition

    The safety of the T cell infusion at the maximum tolerated dose determined in Phase 1, and further characterized in Phase 2, will be described

    Measured: Initial CAR T cell infusion through 30 days post infusion

  2. Number of Participants With an MRD Negative Complete Remission After Initial CAR T Cell Infusion

    The efficacy of the T cell infusion will be estimated based on the number of participants who have an MRD negative bone marrow aspirate following the initial T cell infusion

    Measured: Initial CAR T cell infusion through Day 63 post infusion assessment (+/- 14 days)

  3. Number of Participants Who Have a Releasable Cell Product Generated

    The feasibility of manufacturing and releasing T cell products from pediatric and young adult patients with CD19+ relapsed or refractory leukemia

    Measured: Up to 28 days per manufacturing attempt

Secondary outcomes (3)
  1. Persistence of Functional CD19 CAR+ T Cells

    Participants will be followed for 63 days to determine if the transferred T cells remain detectable in the blood and bone marrow

    Measured: Initial CAR T Infusion through Day 63 post infusion assessment (+/- 14 days)

  2. Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T Infusion

    Measured: Initial CAR T Infusion through Day 63 post infusion assessment (+/- 14 days)

  3. Number of Participants That Received Cetuximab Who Have T Cells Successfully Ablated

    The efficacy of cetuximab to ablate the T cells will be measured by loss of detection of T cells and any associated toxicities as well as facilitating B cell recovery.

    Measured: 3 years

Timeline

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

  1. First posted7 Jan 2014 (estimated)
  2. Study start11 Feb 2014
  3. Primary completion10 Aug 2021
  4. Study completionJul 2036 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
167 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02028455
Sponsor's protocol ID
PLAT-02
Status last verified
Aug 2026
First submitted
3 Jan 2014
Last updated
6 Aug 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Annesley C, Seidel K, Wu Q, Summers C, Wayne AS, Pulsipher MA, Agrawal AK, Brown CT, Mgebroff S, Lindgren C, Rawlings-Rhea S, Huang W, Wilson AL, Jensen MC, Park JR, Gardner RA. Outcomes of PLAT-02 and PLAT-03: evaluating CD19 CAR T-cell therapy and CD19-expressing T-APC support in pediatric B-ALL. Blood. 2025 Aug 14;146(7):789-801. doi: 10.1182/blood.2025028359.

    PubMed 40233328
  • Ceppi F, Wilson AL, Annesley C, Kimmerly GR, Summers C, Brand A, Seidel K, Wu QV, Beebe A, Brown C, Mgebroff S, Lindgren C, Rawlings-Rhea SD, Huang W, Pulsipher MA, Wayne AS, Park JR, Jensen MC, Gardner RA. Modified Manufacturing Process Modulates CD19CAR T-cell Engraftment Fitness and Leukemia-Free Survival in Pediatric and Young Adult Subjects. Cancer Immunol Res. 2022 Jul 1;10(7):856-870. doi: 10.1158/2326-6066.CIR-21-0501.

    PubMed 35580141
  • Ernst M, Oeser A, Besiroglu B, Caro-Valenzuela J, Abd El Aziz M, Monsef I, Borchmann P, Estcourt LJ, Skoetz N, Goldkuhle M. Chimeric antigen receptor (CAR) T-cell therapy for people with relapsed or refractory diffuse large B-cell lymphoma. Cochrane Database Syst Rev. 2021 Sep 13;9(9):CD013365. doi: 10.1002/14651858.CD013365.pub2.

    PubMed 34515338
  • Finney OC, Brakke HM, Rawlings-Rhea S, Hicks R, Doolittle D, Lopez M, Futrell RB, Orentas RJ, Li D, Gardner RA, Jensen MC. CD19 CAR T cell product and disease attributes predict leukemia remission durability. J Clin Invest. 2019 Mar 12;129(5):2123-2132. doi: 10.1172/JCI125423. Print 2019 May 1.

    PubMed 30860496
  • Gardner RA, Finney O, Annesley C, Brakke H, Summers C, Leger K, Bleakley M, Brown C, Mgebroff S, Kelly-Spratt KS, Hoglund V, Lindgren C, Oron AP, Li D, Riddell SR, Park JR, Jensen MC. Intent-to-treat leukemia remission by CD19 CAR T cells of defined formulation and dose in children and young adults. Blood. 2017 Jun 22;129(25):3322-3331. doi: 10.1182/blood-2017-02-769208. Epub 2017 Apr 13.

    PubMed 28408462
  • Gardner R, Wu D, Cherian S, Fang M, Hanafi LA, Finney O, Smithers H, Jensen MC, Riddell SR, Maloney DG, Turtle CJ. Acquisition of a CD19-negative myeloid phenotype allows immune escape of MLL-rearranged B-ALL from CD19 CAR-T-cell therapy. Blood. 2016 May 19;127(20):2406-10. doi: 10.1182/blood-2015-08-665547. Epub 2016 Feb 23.

    PubMed 26907630

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Patient Derived CD19 specific CAR T cells also expressing an EGFRt in people with CD19+ Acute Leukemia, run by Seattle Children's Hospital at 3 sites 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 35 inclusion and 0 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 6 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 3 sites 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Mar 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity Definition (time frame: Initial CAR T cell infusion through 30 days post infusion). 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 NCT02028455, which also lists 6 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 6 Aug 2026.

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Clinical Trial NCT02028455 | AzTrials