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

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

Status
Active, not recruiting
Phases
Phase 1Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT02028455
Enrollment
167
Registered
2014-01-07
Start date
2014-02-11
Completion date
2036-07-01
Last updated
2026-08-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

CD19+ Acute Leukemia

Keywords

pediatric, young adult, acute lymphoblastic leukemia, CD19, leukemia, Chimeric Antigen Receptor, T cell

Brief 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). 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.

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.

Interventions

BIOLOGICALPatient Derived CD19 specific CAR T cells also expressing an EGFRt

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

Sponsors

Seattle Children's Hospital
Lead SponsorOTHER

Study design

Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
1 Years to 26 Years
Healthy volunteers
No

Inclusion criteria

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) * 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.

Design outcomes

Primary

MeasureTime frameDescription
Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionInitial CAR T cell infusion through 30 days post infusionThe safety of the T cell infusion at the maximum tolerated dose determined in Phase 1, and further characterized in Phase 2, will be described
Number of Participants With an MRD Negative Complete Remission After Initial CAR T Cell InfusionInitial CAR T cell infusion through Day 63 post infusion assessment (+/- 14 days)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
Number of Participants Who Have a Releasable Cell Product GeneratedUp to 28 days per manufacturing attemptThe feasibility of manufacturing and releasing T cell products from pediatric and young adult patients with CD19+ relapsed or refractory leukemia

Secondary

MeasureTime frameDescription
Persistence of Functional CD19 CAR+ T CellsInitial CAR T Infusion through Day 63 post infusion assessment (+/- 14 days)Participants will be followed for 63 days to determine if the transferred T cells remain detectable in the blood and bone marrow
Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionInitial CAR T Infusion through Day 63 post infusion assessment (+/- 14 days)
Number of Participants That Received Cetuximab Who Have T Cells Successfully Ablated3 yearsThe 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.

Countries

United States

Contacts

STUDY_CHAIRColleen Annesley, MD

Seattle Children's Hospital

Participant flow

Participants by arm

ArmCount
Phase 1-Cohort 1A
This phase 1 cohort received Patient Derived CD19 specific CAR T cells at a dose of 5x10\^5 CAR T cells/kg
7
Phase 1-Cohort 1B
This phase 1 cohort received Patient Derived CD19 specific CAR T cells at a dose of 1x10\^6 CAR T cells/kg
12
Phase 1-Cohort 1C
This phase 1 cohort received Patient Derived CD19 specific CAR T cells at a dose of 5x10\^6 CAR T cells/kg
7
Phase 1-Cohort 1D
This phase 1 cohort received Patient Derived CD19 specific CAR T cells at a dose of 1x10\^7 CAR T cells/kg
5
Phase 1-Cohort 1F1
This phase 1 cohort received Patient Derived CD19 specific CAR T cells at a dose of 5x10\^5 CAR T cells/kg following prescribed lymphodepletion with fludarabine and cyclophosphamide
6
Phase 1-Cohort 1F2
This phase 1 cohort received Patient Derived CD19 specific CAR T cells at a dose of 1x10\^6 CAR T cells/kg following prescribed lymphodepletion with fludarabine and cyclophosphamide
6
Phase 1 - Not Treated
This phase 1 cohort did not receive Patient Derived CD19 specific CAR T cells
4
Phase 2
The phase 2 cohort received Patient Derived CD19 specific CAR T cells following lymphodepletion if indicated
120
Total167

Baseline characteristics

CharacteristicPhase 1-Cohort 1BPhase 1-Cohort 1CPhase 1-Cohort 1DPhase 1-Cohort 1F1Phase 1-Cohort 1F2Phase 1-Cohort 1APhase 1 - Not TreatedPhase 2Total
Age, Continuous9 years15 years6 years10 years13.5 years19 years11 years13 years13 years
Diagnosis
Leukemia
12 Participants7 Participants5 Participants6 Participants6 Participants7 Participants4 Participants106 Participants153 Participants
Diagnosis
Non Hodgkin's Lymphoma
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants14 Participants14 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
4 Participants2 Participants2 Participants0 Participants1 Participants1 Participants1 Participants38 Participants49 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
8 Participants5 Participants3 Participants5 Participants4 Participants6 Participants2 Participants72 Participants105 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants1 Participants1 Participants0 Participants1 Participants10 Participants13 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
0 Participants1 Participants1 Participants0 Participants1 Participants1 Participants0 Participants6 Participants10 Participants
Race (NIH/OMB)
Black or African American
1 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants4 Participants5 Participants
Race (NIH/OMB)
More than one race
1 Participants1 Participants0 Participants0 Participants0 Participants0 Participants0 Participants4 Participants6 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants1 Participants1 Participants
Race (NIH/OMB)
Unknown or Not Reported
2 Participants1 Participants2 Participants1 Participants2 Participants1 Participants1 Participants39 Participants49 Participants
Race (NIH/OMB)
White
8 Participants4 Participants2 Participants5 Participants3 Participants5 Participants3 Participants66 Participants96 Participants
Sex: Female, Male
Female
3 Participants3 Participants5 Participants3 Participants4 Participants4 Participants1 Participants40 Participants63 Participants
Sex: Female, Male
Male
9 Participants4 Participants0 Participants3 Participants2 Participants3 Participants3 Participants80 Participants104 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
EG002
affected / at risk
EG003
affected / at risk
EG004
affected / at risk
EG005
affected / at risk
EG006
affected / at risk
deaths
Total, all-cause mortality
4 / 79 / 123 / 75 / 53 / 62 / 640 / 103
other
Total, other adverse events
7 / 712 / 127 / 75 / 56 / 66 / 6103 / 103
serious
Total, serious adverse events
6 / 710 / 125 / 75 / 52 / 64 / 648 / 103

Outcome results

Primary

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

Time frame: Initial CAR T cell infusion through 30 days post infusion

Population: Phase 1 cohorts: Toxicity-evaluable leukemia patients who received CAR T cells; Phase 2 cohort: Toxicity-evaluable leukemia patients treated with the Phase 2 dose, 1 x 10\^6 CAR T cells/kg, following lymphodepletion with fludarabine and cyclophosphamide

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Phase 1 - Cohort 1ANumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition1 Participants
Phase 1 - Cohort 1ANumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition6 Participants
Phase 1-Cohort 1BNumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition1 Participants
Phase 1-Cohort 1BNumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition11 Participants
Phase 1-Cohort 1CNumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition2 Participants
Phase 1-Cohort 1CNumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition5 Participants
Phase 1-Cohort 1DNumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition2 Participants
Phase 1-Cohort 1DNumber of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition3 Participants
Phase 1-Cohort 1F1Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition0 Participants
Phase 1-Cohort 1F1Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition6 Participants
Phase 1-Cohort 1F2Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition1 Participants
Phase 1-Cohort 1F2Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition5 Participants
Phase 2Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who experienced an AE meeting the Dose-Limiting Toxicity definition17 Participants
Phase 2Number of Participants Who Experienced an Adverse Event Meeting the Dose-Limiting Toxicity DefinitionNumber of participants who did not experience an AE meeting the Dose-Limiting Toxicity definition64 Participants
Primary

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

Time frame: Up to 28 days per manufacturing attempt

Population: Participants evaluable for manufacturing feasibility.

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Phase 1 - Cohort 1ANumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured7 Participants
Phase 1 - Cohort 1ANumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured0 Participants
Phase 1-Cohort 1BNumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured12 Participants
Phase 1-Cohort 1BNumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured0 Participants
Phase 1-Cohort 1CNumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured7 Participants
Phase 1-Cohort 1CNumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured0 Participants
Phase 1-Cohort 1DNumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured5 Participants
Phase 1-Cohort 1DNumber of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured0 Participants
Phase 1-Cohort 1F1Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured6 Participants
Phase 1-Cohort 1F1Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured0 Participants
Phase 1-Cohort 1F2Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured6 Participants
Phase 1-Cohort 1F2Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured0 Participants
Phase 2Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured2 Participants
Phase 2Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured1 Participants
Phase 2Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom a releasable CAR T cell product was manufactured116 Participants
Phase 2Number of Participants Who Have a Releasable Cell Product GeneratedNumber of participants for whom no releasable CAR T cell product was manufactured3 Participants
Primary

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

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

Population: Efficacy-evaluable leukemia patients in Phase 2, plus Phase 1 patients treated at the Phase 2 dose in cohort 1F2, who received SCRIv1 or SCRIv1.5 CAR T cells following prescribed lymphodepletion with fludarabine and cyclophosphamide

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Phase 1 - Cohort 1ANumber of Participants With an MRD Negative Complete Remission After Initial CAR T Cell InfusionNumber of Participants with an MRD Negative Complete Remission after Initial CAR T Infusion63 Participants
Phase 1 - Cohort 1ANumber of Participants With an MRD Negative Complete Remission After Initial CAR T Cell InfusionNumber of Participants without an MRD Negative Complete Remission after Initial CAR T Infusion8 Participants
Secondary

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.

Time frame: 3 years

Population: Number of subjects receiving cetuximab on study for ablation of CAR T cells

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Phase 1 - Cohort 1ANumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells0 Participants
Phase 1 - Cohort 1ANumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Phase 1-Cohort 1BNumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells0 Participants
Phase 1-Cohort 1BNumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Phase 1-Cohort 1CNumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells0 Participants
Phase 1-Cohort 1CNumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Phase 1-Cohort 1DNumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells0 Participants
Phase 1-Cohort 1DNumber of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Phase 1-Cohort 1F1Number of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells0 Participants
Phase 1-Cohort 1F1Number of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Phase 1-Cohort 1F2Number of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells0 Participants
Phase 1-Cohort 1F2Number of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Phase 2Number of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants receiving cetuximab on study for ablation of CAR T cells2 Participants
Phase 2Number of Participants That Received Cetuximab Who Have T Cells Successfully AblatedNumber of participants whose CAR T cells were successfully ablated by cetuximab administration0 Participants
Secondary

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

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

Population: Number of participants with a history of prior allogeneic HSCT (hematopoietic stem cell transplantation) who received CAR T infusion

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Phase 1 - Cohort 1ANumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion6 Participants
Phase 1 - Cohort 1ANumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion1 Participants
Phase 1-Cohort 1BNumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion7 Participants
Phase 1-Cohort 1BNumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion0 Participants
Phase 1-Cohort 1CNumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion4 Participants
Phase 1-Cohort 1CNumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion0 Participants
Phase 1-Cohort 1DNumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion3 Participants
Phase 1-Cohort 1DNumber of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion0 Participants
Phase 1-Cohort 1F1Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion4 Participants
Phase 1-Cohort 1F1Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion0 Participants
Phase 1-Cohort 1F2Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion3 Participants
Phase 1-Cohort 1F2Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion0 Participants
Phase 2Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects without recrudescence or development of Acute GVHD symptoms post CAR T infusion36 Participants
Phase 2Number of Participants With Recrudescence or Development of Acute GVHD (Graft Versus Host Disease) Symptoms Post CAR T InfusionNumber of subjects with recrudescence or development of Acute GVHD symptoms post CAR T infusion0 Participants
Secondary

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

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

Population: Efficacy-evaluable leukemia patients in Phase 2, plus Phase 1 patients treated at the Phase 2 dose in Cohort 1F2, who received SCRIv1 or SCRIv1.5 CAR T cells following prescribed lymphodepletion with fludarabine and cyclophosphamide and who were followed for loss of persistence through the Day 63 assessment window. Excludes one participant with insufficient samples collected to determine Day 63 persistence status.

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Phase 1 - Cohort 1APersistence of Functional CD19 CAR+ T CellsNumber of participants without functional CAR T cell persistence through the Day 63 study assessment28 Participants
Phase 1 - Cohort 1APersistence of Functional CD19 CAR+ T CellsNumber of participants with functional CAR T cell persistence through the Day 63 study assessment42 Participants

Source: ClinicalTrials.gov · Data processed: Aug 7, 2026