B Cell LeukemiaPhase 1NCT01683279
A Pediatric Trial of Genetically Modified Autologous T Cells Directed Against CD19 for Relapsed CD19+ Acute Lymphoblastic Leukemia
A Phase 1 study testing Autologous CD19 CAR+ EGFTt + T cells in people with B Cell Leukemia, run by Seattle Children's Hospital at 1 site in United States.
Trial summary
Patients with relapsed leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use a patient's own T cells, which can be genetically modified to expresses 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
- B Cell Leukemia
- What is being tested?
- Autologous CD19 CAR+ EGFTt + T cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Mar 2012to 7 Jan 2030
- 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 1 year to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- CD19+ Leukemia in 1st marrow relapse with MRD at the end of 1st month of re-induction
- CD19+ Leukemia in 2nd or greater relapse
- CD19+ Leukemia with indication for HCT, but has contraindication
- Age between 1 and 26 years of age
- Karnofsky of >50 or Lansky >50
- Life Expectancy >12 weeks
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- Able to tolerate a blood draw of 4-6mL/kg
- Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy
- absolute lymphocyte count of >/=750 cell/mm3 or >/=500 is >20kg
- creatinine clearance or radioisotope GFR >/= 70mL/min/1.73m2 OR normal serum creatinine based on age/gender
- total bilirubin </= 1.5x upper limit normal OR direct bilirubin </= 1.5mg/dl
- ALT </= 3x upper limit normal
- corrected QTc <450msec of ECG
- Shortening Fraction >28% by ECHO or Ejection Fraction >50% by MUGA
- Documented negative HIV, Hep B and Hep C
- Agree to long-term follow up for up to 15 years if they receive T cell infusion
Who cannot join
- Philadelphia Positive Leukemia
- Prior Allogeneic Stem Cell Transplant
- CNS 2 or 3
- prior cellular immunotherapy with chimeric antigen receptor modified T cells
- fully humanized antibodies within three half lives
- systemic corticosteroids within 7 days of enrollment
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- requires supplemental oxygen or has a chest X-ray with an infectious process
- CNS pathology (seizure disorder, paresis, aphasia, cerebrovascular ischemia/hemorrhage, severe brain injuries, dementia, cerebellar disease, organic brain syndrome, psychosis, coordination or movement disorder)
- Pregnant or breastfeeding women. Female participant of reproductive age must have a negative pregnancy test and agree to contraception for 1 year after T cell infusion.
- Active Malignancy other than CD19+ Leukemia
- Active severe infection defined as a positive blood culture within 48 hours of study enrollment or a fever >38.2C AND clinical signs of infection within 48 hours of study enrollment
- Patient has a concurrent medical condition, that in the opinion of the protocol PI or designee, would prevent the patient from undergoing protocol-based therapy.
- Trisomy 21
- Primary immunodeficiency/bone marrow failure syndrome
Trial locations and contacts
1 site in 1 country.
United States1 site
Seattle Children's Hospital
Seattle, Washington, 98105
About this study
Official title: Pediatric Leukemia Adoptive Therapy (PLAT)-01: A Phase 1 Feasibility and Safety Study of Cellular Immunotherapy for Relapsed Pediatric CD19+ Acute Lymphoblastic Leukemia Using Autologous T-cells Lentivirally Transduced To Express a CD19-Specific Chimeric Antigen Receptor
Patients with relapsed leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use a patient's own T cells, which can be genetically modified to expresses a chimeric antigen receptor(CAR). The CAR enables the T cell to recognize and kill the leukemic cells though the recognition of CD19, a protein expressed on the surface of the majority of pediatric ALL. This is a phase I study designed to determine the maximum tolerated dose of the CAR+ T cells and define the toxicity of the treatment. As a secondary aim, we will be looking at the efficacy of the T cells on eradicating the patient's leukemic cells.
Read the detailed description
Upon meeting the eligibility requirements and enrolling on study, subjects will undergo a blood draw to obtain the T cells for the generation of the CD19 CAR+ T cells. The T cells are isolated from the blood, transduced with a lentivirus to express the CD19 CAR, 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 will be admitted to the hospital to receive 2 days of cyclophosphamide for lymphodepletion and reduction of disease burden. Several days later, the subject will receive an infusion of the CAR+ T cells.
Following treatment with the CAR+ T cells, subjects will be intensely followed for 6 weeks with serial blood testing and re-evaluation of disease status with bone marrow aspirates. After 6 weeks, the subjects clinical care will be resumed by their primary oncologist, and it is possible that they would receive additionally chemotherapy or a stem cell transplant.
Upon completion of the study, subjects will be followed at least annually with a either a medical history, physical exam and blood tests or a phone call/questionnaire for 15 years. 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.
- Conditions
- B Cell Leukemia
- Being tested
- Autologous CD19 CAR+ EGFTt + T cells
- Keywords
- pediatric, acute lymphoblastic leukemia, CD19, Chimeric Antigen Receptor
- Medical subject headings
- Leukemia, B-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous CD19 CAR+ EGFTt + T cellsBiological
Autologous T cell modified to express a CD19 specific CAR and a truncated EGFRt tag
| Study group | Role | What they receive |
|---|---|---|
| CAR+ T cells | Experimental | Subjects will receive two days of cyclophosphamide for a total of 3g/m^2 followed several days later by a single dose of Autologous CD19 CAR+ EGFTt + T cells Receives: Biological: Autologous CD19 CAR+ EGFTt + T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participant with Adverse Events
The safety of the T cell infusion will be described and the maximum tolerated dose determined.
Measured: 42 days
Secondary outcomes (2)
Persistence of the CD19 CAR+ T cells
Patients will be followed for 42 days to determine if the transferred T cells remain detectable in the blood and bone marrow
Measured: 42 days
Determine if there is anti-leukemic activity of the CD19 CAR+ T cells
Patients will have their bone marrow assessed following the T cell infusion to determine if their disease responded to the treatment
Measured: 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2012 (estimated)
- Study start25 Mar 2012
- Primary completion7 Jan 2015
- Study completion7 Jan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for B Cell 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
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
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
- Investigators
- Colleen Annesley, MD, Study chair, Seattle Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01683279
- Sponsor's protocol ID
- PLAT-01
- Status last verified
- Jun 2026
- First submitted
- 7 Sep 2012
- Last updated
- 5 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pediatric Leukemia Adoptive Therapy (PLAT)-01: A Phase 1 Feasibility and Safety Study of Cellular Immunotherapy for Relapsed Pediatric CD19+ Acute Lymphoblastic Leukemia Using Autologous T-cells Lentivirally Transduced To Express a CD19-Specific Chimeric Antigen Receptor
- ClinicalTrials.gov ID
- NCT01683279
- Acronym / sponsor ID
- PLAT-01
- Conditions
- B Cell Leukemia
- Keywords
- pediatric, acute lymphoblastic leukemia, CD19, Chimeric Antigen Receptor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Seattle Children's HospitalUniversity, hospital or other
- Study start
- 25 Mar 2012 (actual)
- Primary completion
- 7 Jan 2015 (actual)
- Study completion
- 7 Jan 2030 (estimated)
- First posted
- 11 Sep 2012 (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.
Patients with relapsed leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use a patient's own T cells, which can be genetically modified to expresses a chimeric antigen receptor(CAR). The CAR enables the T cell to recognize and kill the leukemic cells though the recognition of CD19, a protein expressed on the surface of the majority of pediatric ALL. This is a phase I study designed to determine the maximum tolerated dose of the CAR+ T cells and define the toxicity of the treatment. As a secondary aim, we will be looking at the efficacy of the T cells on eradicating the patient's leukemic cells.
Detailed description
Upon meeting the eligibility requirements and enrolling on study, subjects will undergo a blood draw to obtain the T cells for the generation of the CD19 CAR+ T cells. The T cells are isolated from the blood, transduced with a lentivirus to express the CD19 CAR, 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 will be admitted to the hospital to receive 2 days of cyclophosphamide for lymphodepletion and reduction of disease burden. Several days later, the subject will receive an infusion of the CAR+ T cells.
Following treatment with the CAR+ T cells, subjects will be intensely followed for 6 weeks with serial blood testing and re-evaluation of disease status with bone marrow aspirates. After 6 weeks, the subjects clinical care will be resumed by their primary oncologist, and it is possible that they would receive additionally chemotherapy or a stem cell transplant.
Upon completion of the study, subjects will be followed at least annually with a either a medical history, physical exam and blood tests or a phone call/questionnaire for 15 years. 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAR+ T cells | Experimental | Biological: Autologous CD19 CAR+ EGFTt + T cellsSubjects will receive two days of cyclophosphamide for a total of 3g/m^2 followed several days later by a single dose of Autologous CD19 CAR+ EGFTt + T cells |
Who may qualify
- Ages
- 1 year to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- CD19+ Leukemia in 1st marrow relapse with MRD at the end of 1st month of re-induction
- CD19+ Leukemia in 2nd or greater relapse
- CD19+ Leukemia with indication for HCT, but has contraindication
- Age between 1 and 26 years of age
- Karnofsky of >50 or Lansky >50
Key exclusion criteria (14 listed)
- Philadelphia Positive Leukemia
- Prior Allogeneic Stem Cell Transplant
- CNS 2 or 3
- prior cellular immunotherapy with chimeric antigen receptor modified T cells
- fully humanized antibodies within three half lives
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 |
|---|---|---|
| Autologous CD19 CAR+ EGFTt + T cellsAutologous T cell modified to express a CD19 specific CAR and a truncated EGFRt tag | Biological | CAR+ T cells |
Primary outcome
Number of Participant with Adverse Events
Time frame: 42 days
The safety of the T cell infusion will be described and the maximum tolerated dose determined.
2 secondary outcomes
Persistence of the CD19 CAR+ T cells
Time frame: 42 days
Determine if there is anti-leukemic activity of the CD19 CAR+ T cells
Time frame: 42 days
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 5 Jun 2026.
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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.
Colleen Annesley, MD study chair
Seattle Children's Hospital
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 Autologous CD19 CAR+ EGFTt + T cells in people with B Cell Leukemia, 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 16 inclusion and 14 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 5 Jun 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?
Number of Participant with Adverse Events (time frame: 42 days). 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 5 Jun 2026.