A Study Evaluating the Safety and Efficacy of Brexucabtagene Autoleucel (KTE-X19) in Adult Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (ZUMA-3) (ZUMA-3)

• ClinicalTrials.gov Identifier: NCT02614066
• Recruitment Status: Completed
• First Posted: November 25, 2015
• Results First Posted: November 29, 2021
• Last Update Posted: November 18, 2023
• Sponsor: Kite, A Gilead Company
• Information provided by (Responsible Party): Gilead Sciences ( Kite, A Gilead Company )

• Study Type: Interventional
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
• Interventions: Biological: brexucabtagene autoleucel; Drug: Cyclophosphamide; Drug: Fludarabine
• Enrollment: 125

Participant Flow

Recruitment Details	Participants were enrolled at study sites in Canada, France, Germany, Netherlands and United States. The first participant was screened on 07 March 2016. The last primary analysis study visit occurred on 09 September 2020.
Pre-assignment Details	173 participants were screened. Bridging therapy was recommended for all participants particularly those participants with high disease burden at baseline (M3 marrow [> 25% leukemic blasts] or ≥ 1,000 blasts/mm^3 in the peripheral circulation) to control participant's disease post apheresis/enrollment and prior to conditioning chemotherapy.

Arm/Group Title	Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 0.5 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description	Participants with relapsed or refractory B-precursor acute lymphoblastic leukemia (r/r B-ALL) received conditioning chemotherapy (fludarabine 25 mg/m^2 intravenously [IV] over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel (KTE-X19) chimeric antigen receptor (CAR) transduced autologous T cells administered IV at a target dose of 2 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 2 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 0.5 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 0.5 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Period Title: Overall Study
Started	6	28	20	71
Completed	0	0	0	0
Not Completed	6	28	20	71
Reason Not Completed
Initiated KTE-X19 and Died	5	12	11	20
Full Consent Withdrawn	0	1	0	3
Lost to Follow-up	0	1	0	0
Still on-study	1	9	5	32
Enrolled But Did Not Initiate KTE-X19	0	5	4	16

Baseline Characteristics

Arm/Group Title		Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 0.5 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Total
Arm/Group Description		Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 2 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 2 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 0.5 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 0.5 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Total of all reporting groups
Overall Number of Baseline Participants		6	23	16	55	100
Baseline Analysis Population Description		The Safety Analysis Set included all enrolled participants treated with any dose of brexucabtagene autoleucel.
Age, Customized; Measure Type: Count of Participants; Unit of measure: Participants	Number Analyzed	6 participants	23 participants	16 participants	55 participants	100 participants
< 65 Years		6	19	13	47	85
≥ 65 Years		0	4	3	8	15
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	6 participants	23 participants	16 participants	55 participants	100 participants
	Female	1	14	8	22	45
	Male	5	9	8	33	55
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants
Ethnicity	Number Analyzed	6 participants	23 participants	16 participants	55 participants	100 participants
	Hispanic or Latino	3	11	3	11	28
	Not Hispanic or Latino	3	12	13	42	70
	Missing	0	0	0	2	2
Race/Ethnicity, Customized; Measure Type: Count of Participants; Unit of measure: Participants
Race	Number Analyzed	6 participants	23 participants	16 participants	55 participants	100 participants
	American Indian or Alaska Native	0	0	0	1	1
	Asian	1	2	0	3	6
	Black or African American	0	0	0	1	1
	White	4	19	16	37	76
	Other	1	1	0	9	11
	Missing	0	0	0	4	4
	Native Hawaiian or Other Pacific Islander	0	1	0	0	1
Region of Enrollment; Measure Type: Number; Unit of measure: Participants	Number Analyzed	6 participants	23 participants	16 participants	55 participants	100 participants
France		0	0	0	10	10
Germany		0	0	0	3	3
Netherlands		0	0	0	1	1
United States		6	23	16	41	86

Outcome Measures

1. Primary Outcome

Title	Phase 1: Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs)
Description	DLT is drug-related events with onset within first 28 days following infusion: Grade (GR) 4 hematologic toxicity lasting more than 30 days (except lymphopenia) if not attributable to underlying disease; All drug-related GR 3 lasting for > 7 days or 4 non-hematologic toxicities regardless of duration (except: aphasia/dysphasia or confusion/cognitive disturbance which resolves to at least GR 1/baseline within 2 weeks or baseline within 4 weeks, fever GR 3/ 4, immediate hypersensitivity reactions within 2 hours of drug infusion that are reversible ≤ GR 2 within 24 hours, renal toxicity which requires dialysis for ≤ 7 days, intubation for airway protection if ≤ 7 days, tumor lysis syndrome, GR 3 liver function test elevation, provided there is resolution to ≤ GR 2 within 14 days, GR 4 transient serum hepatic enzyme abnormalities provided there is resolution to ≤ GR 3 within < 72 hours, hypogammaglobulinemia GR 3/ 4 and GR 3 nausea and/or anorexia).
Time Frame	First infusion date of brexucabtagene autoleucel up to 28 days. Participants were evaluated in specified period but GR4 hematologic toxicity (specified in description) having onset in this period were further observed for 30 days for confirmation

Outcome Measure Data

Analysis Population Description

DLT-Evaluable Analysis Set included first 3 participants in Phase 1 treated with target brexucabtagene autoleucel dose and followed for at least 28 days. Only participants from Phase 1: 2 X 10^6 Anti-CD19 CAR T Cells/kg were pre-specified to be assessed for this Outcome Measure.

Arm/Group Title	Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 2 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 2 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	3
Measure Type: Number; Unit of Measure: percentage of participants
	0

2. Primary Outcome

Title	Phase 2: Overall Complete Remission (OCR) Rate (Complete Remission [CR]+ Complete Remission With Incomplete Hematologic Recovery [CRi]) as Assessed Per Independent Review
Description	OCR rate: percentage of participants achieving CR+CRi. CR: ≤5% blasts by morphology in bone marrow (BM); absolute neutrophil count (ANC) ≥1000 and platelets (Plt) ≥100000 in peripheral blood (PB); central nervous system extramedullary disease (CNS EMD) of CNS-1 (no detectable leukemia in cerebrospinal fluid [CSF]); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative positron emission tomography (PET) baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses >1.5 cm in greatest transverse diameter [GTD] at baseline must have regressed to ≤l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and >1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. CRi: all CR criteria except in PB ANC ≥1000 and Plt <100000 or ANC <1000 and Plt ≥100000.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Modified Intent-to-Treat (mITT) Analysis Set included all enrolled participants treated with brexucabtagene autoleucel in Phase 2.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	70.9; (57 to 82)

3. Secondary Outcome

Title	Phase 2: Minimum Residual Disease (MRD) Negative Remission Rate
Description	MRD was assessed utilizing multicolor flow cytometry to detect residual cancerous cells with a sensitivity of 10^-4. MRD negative remission was defined as MRD < 10^-4 threshold. Percentage of participants with MRD negative remission was reported.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	76; (63 to 87)

4. Secondary Outcome

Title	Phase 2: Complete Remission (CR) Rate Per Independent Review
Description	CR: ≤ 5% blasts by morphology in BM; ANC ≥ 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses > 1.5 cm in GTD at baseline must have regressed to ≤ l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and > 1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. Percentage of participants with CR was reported.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	56.4; (42 to 70)

5. Secondary Outcome

Title	Phase 2: Complete Remission With Incomplete Hematologic Recovery (CRi) Rate Per Independent Review
Description	CRi: ≤ 5% blasts by morphology in BM; ANC ≥ 1000 and Plt < 100000 or ANC < 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses > 1.5 cm in GTD at baseline must have regressed to ≤ l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and > 1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. Percentage of participants with CRi was reported.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	14.5; (6 to 27)

6. Secondary Outcome

Title	Phase 2: Duration of Remission (DOR) Per Independent Review
Description	DOR was defined as the time from first CR or CRi to relapse or any death in the absence of documented relapse. CR and CRi are defined in Outcome Measures 4 and 5. Relapse: ≤ 5% blasts by morphology in BM; or circulating leukemia present in PB; or CNS EMD of CNS-2 (detectable CSF blast cells in a sample of CSF with < 5 white blood cells [WBCs] per mm^3 with neurological changes) or CNS-3 (detectable CSF blast cells in a sample of CSF with ≥ 5 WBCs per mm^3 with or without neurological changes); or progressive disease (PD): at least one of the following (≥ 50% increase from nadir in the sum of the products of at least two lymph nodes, or if a single node is involved at least a 50% increase in the product of the diameters of this one node; at least a 50% increase in the longest diameter of any single previously identified node more than 1 cm in its short axis; ≥ 50% increase in size of splenic, hepatic or any other non-nodal lesion). Kaplan-Meier (KM) estimates was used for analyses.
Time Frame	From first CR or CRi (Phase 2) to relapse/death or data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Median (95% Confidence Interval); Unit of Measure: months
	12.8 [1]; (8.7 to NA)

[1]

Upper limit of confidence interval was not estimable due to insufficient number of events.

7. Secondary Outcome

Title	Phase 2: OCR Rate (CR + CRi) Per Investigator Review
Description	OCR rate: percentage of participants achieving CR+CRi. CR: ≤ 5% blasts by morphology in BM; ANC ≥1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses >1.5 cm in GTD at baseline must have regressed to ≤l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and >1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions. CRi: all CR criteria except in PB ANC ≥1000 and Plt <100000 or ANC <1000 and Plt ≥100000.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	72.7; (59 to 84)

8. Secondary Outcome

Title	Phase 2: Percentage of Participants With Allogeneic Stem Cell Transplant (Allo-SCT)
Description	[Not Specified]
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Unit of Measure: percentage of participants
	18

9. Secondary Outcome

Title	Phase 2: MRD Negative Rate Among Complete Remission (CR) Participants
Description	Percentage of participants with MRD negative remission among CR participants was reported. MRD was assessed utilizing multicolor flow cytometry to detect residual cancerous cells with a sensitivity of 10^-4. MRD negative remission was defined as MRD < 10^-4 threshold. CR: ≤5% blasts by morphology in BM; ANC ≥ 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses > 1.5 cm in GTD at baseline must have regressed to ≤ l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and > 1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set with CR were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	31
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	97; (83 to 100)

10. Secondary Outcome

Title	Phase 2: MRD Negative Rate Among Complete Remission With Incomplete Hematologic Recovery (CRi) Participants
Description	Percentage of participants with MRD negative remission among CRi participants was reported. MRD was assessed utilizing multicolor flow cytometry to detect residual cancerous cells with a sensitivity of 10^-4. MRD negative remission was defined as MRD < 10^-4 threshold. CRi: ≤ 5% blasts by morphology in BM; ANC ≥ 1000 and Plt < 100000 or ANC < 1000 and Plt ≥ 100000 in PB; CNS EMD of CNS-1 (no detectable leukemia in CSF); Non-CNS EMD: if baseline EMD present (images must show CR), if no baseline EMD (then images not required), but if performed should show negative PET baseline, baseline lesions must show CR as disappearance of measurable and nonmeasurable nodal lesions (Nodal masses >1.5 cm in GTD at baseline must have regressed to ≤l.5 cm GTD, nodes that were 1.1 to 1.5 cm [long axis] and >1.0 cm [short axis] must have decreased to 1.0 cm in their short axis, spleen and/or liver must be normal size, if tested) and no new lesions.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set with CRi were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	8
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
	100; (63 to 100)

11. Secondary Outcome

Title	Phase 2: Overall Survival (OS)
Description	OS was defined as the time from brexucabtagene autoleucel infusion to the date of death from any cause. Participants who had not died by the analysis data cutoff date were censored at their last contact date. KM estimates was used for analyses.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to death or data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Median (95% Confidence Interval); Unit of Measure: months
	18.2 [1]; (15.9 to NA)

[1]

Upper limit of confidence interval was not estimable due to insufficient number of events.

12. Secondary Outcome

Title	Phase 2: Relapse-free Survival (RFS)
Description	RFS: time from brexucabtagene autoleucel infusion to date of disease relapse or death from any cause. Participants not meeting criteria for relapse by the analysis data cutoff date were censored at their last evaluable disease assessment date. Participants who had not achieved a CR or CRi at analysis data cutoff were evaluated as an RFS event at Day 0. CR and CRi are defined in Outcome Measures 4 and 5. Relapse is defined in Outcome Measure 6. KM estimates was used for analyses.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to relapse/death or data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in mITT Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Median (95% Confidence Interval); Unit of Measure: months
	11.6; (2.7 to 15.5)

13. Secondary Outcome

Title	Phase 2: Percentage of Participants Experiencing Treatment Emergent Adverse Events (TEAEs)
Description	An AE was any untoward medical occurrence in a participant after brexucabtagene autoleucel infusion, which did not necessarily have a causal relationship with the treatment. An AE could therefore be any unfavorable and/or unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. TEAEs included all AEs with onset on or after initiation of the brexucabtagene autoleucel infusion.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

The Safety Analysis Set included all enrolled participants treated with any dose of brexucabtagene autoleucel.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Unit of Measure: percentage of participants
	100

14. Secondary Outcome

Title	Phase 2: Number of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Increased Parameter Value
Description	Grading categories are determined by Common Terminology Criteria for Adverse Events (CTCAE) version 4.03.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in Safety Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Count of Participants; Unit of Measure: Participants
Hematology: Lymphocytes	1
Hematology: Leukocytes	4
Hematology: Hemoglobin	0
Hematology: Neutrophils	0
Hematology: Platelets	0
Chemistry: Creatinine	4
Chemistry: Glucose	13
Chemistry: Aspartate Aminotransferase	14
Chemistry: Alanine Aminotransferase	17
Chemistry: Bilirubin	5
Chemistry: Alkaline Phosphatase	3
Chemistry: Direct Bilirubin	8
Chemistry: Urate	11
Chemistry: Sodium	1
Chemistry: Potassium	2
Chemistry: Magnesium	3
Chemistry: Calcium	0
Chemistry: Albumin	0
Chemistry: Phosphate	0

15. Secondary Outcome

Title	Phase 2: Number of Participants Experiencing Laboratory Toxicity Grade Shifts to Grade 3 or Higher Resulting From Decreased Parameter Value
Description	Grading categories are determined by CTCAE version 4.03.
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in Safety Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Count of Participants; Unit of Measure: Participants
Hematology: Hemoglobin	42
Hematology: Leukocytes	54
Hematology: Platelets	46
Hematology: Lymphocytes	52
Hematology: Neutrophils	53
Chemistry: Calcium	9
Chemistry: Albumin	5
Chemistry: Phosphate	27
Chemistry: Magnesium	0
Chemistry: Sodium	11
Chemistry: Potassium	7
Chemistry: Glucose	0
Chemistry: Alanine Aminotransferase	0
Chemistry: Alkaline Phosphatase	0
Chemistry: Aspartate Aminotransferase	0
Chemistry: Bilirubin	0
Chemistry: Creatinine	0
Chemistry: Direct Bilirubin	0
Chemistry: Urate	0

16. Secondary Outcome

Title	Phase 2: Percentage of Participants With Anti-KTE-X19 Antibodies
Description	[Not Specified]
Time Frame	First infusion date of brexucabtagene autoleucel (Phase 2) to data cutoff date of 09 September 2020 (Maximum duration: 23 Months)

Outcome Measure Data

Analysis Population Description

Participants in Safety Analysis Set were analyzed.

Arm/Group Title	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed	55
Measure Type: Number; Unit of Measure: percentage of participants
	7

17. Secondary Outcome

Title	Phase 2: Number of Participants With 5-Level European Quality of Life-5 Dimensions (EQ-5D-5L): Health Utility Index Scale
Description	EQ-5D-5L is a self-reported questionnaire used for assessing the overall health status of a participant scoring 5 dimensions of health: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was divided into 5 levels of severity: "No problem", "Slight problems", "Moderate problems", "Severe problems", and "Extreme problems" or "Unable to".
Time Frame	Baseline, Day 28, Month 3, Month 6, Month 9, Month 12

Outcome Measure Data

Analysis Population Description

Participants in Safety Analysis Set with available data were analyzed.

Arm/Group Title		Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:		Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed		51
Measure Type: Count of Participants; Unit of Measure: Participants
Baseline: Mobility (No problem)	Number Analyzed	51 participants
		39
Baseline: Mobility (Slight problem)	Number Analyzed	51 participants
		7
Baseline: Mobility (Moderate problem)	Number Analyzed	51 participants
		4
Baseline: Mobility (Severe problem)	Number Analyzed	51 participants
		1
Baseline: Mobility (Unable to walk)	Number Analyzed	51 participants
		0
Day 28: Mobility (No problem)	Number Analyzed	42 participants
		19
Day 28: Mobility (Slight problem)	Number Analyzed	42 participants
		10
Day 28: Mobility (Moderate problem)	Number Analyzed	42 participants
		9
Day 28: Mobility (Severe problem)	Number Analyzed	42 participants
		1
Day 28: Mobility (Unable to walk)	Number Analyzed	42 participants
		3
Month 3: Mobility (No problem)	Number Analyzed	26 participants
		19
Month 3: Mobility (Slight problem)	Number Analyzed	26 participants
		5
Month 3: Mobility (Moderate problem)	Number Analyzed	26 participants
		1
Month 3: Mobility (Severe problem)	Number Analyzed	26 participants
		0
Month 3: Mobility (Unable to walk)	Number Analyzed	26 participants
		1
Month 6: Mobility (No problem)	Number Analyzed	25 participants
		16
Month 6: Mobility (Slight problem)	Number Analyzed	25 participants
		6
Month 6: Mobility (Moderate problem)	Number Analyzed	25 participants
		2
Month 6: Mobility (Severe problem)	Number Analyzed	25 participants
		0
Month 6: Mobility (Unable to walk)	Number Analyzed	25 participants
		1
Month 9: Mobility (No problem)	Number Analyzed	10 participants
		8
Month 9: Mobility (Slight problem)	Number Analyzed	10 participants
		1
Month 9: Mobility (Moderate problem)	Number Analyzed	10 participants
		0
Month 9: Mobility (Severe problem)	Number Analyzed	10 participants
		0
Month 9: Mobility (Unable to walk)	Number Analyzed	10 participants
		1
Month 12: Mobility (No problem)	Number Analyzed	14 participants
		11
Month 12: Mobility (Slight problem)	Number Analyzed	14 participants
		3
Month 12: Mobility (Moderate problem)	Number Analyzed	14 participants
		0
Month 12: Mobility (Severe problem)	Number Analyzed	14 participants
		0
Month 12: Mobility (Unable to walk)	Number Analyzed	14 participants
		0
Baseline: Self-care (No problem)	Number Analyzed	51 participants
		44
Baseline: Self-care (Slight problem)	Number Analyzed	51 participants
		5
Baseline: Self-care (Moderate problem)	Number Analyzed	51 participants
		1
Baseline: Self-care (Severe problem)	Number Analyzed	51 participants
		1
Baseline: Self-care (Unable to wash or dress)	Number Analyzed	51 participants
		0
Day 28: Self-care (No problem)	Number Analyzed	42 participants
		31
Day 28: Self-care (Slight problem)	Number Analyzed	42 participants
		6
Day 28: Self-care (Moderate problem)	Number Analyzed	42 participants
		1
Day 28: Self-care (Severe problem)	Number Analyzed	42 participants
		2
Day 28: Self-care (Unable to wash or dress)	Number Analyzed	42 participants
		2
Month 3: Self-care (No problem)	Number Analyzed	25 participants
		23
Month 3: Self-care (Slight problem)	Number Analyzed	25 participants
		1
Month 3: Self-care (Moderate problem)	Number Analyzed	25 participants
		1
Month 3: Self-care (Severe problem)	Number Analyzed	25 participants
		0
Month 3: Self-care (Unable to wash or dress)	Number Analyzed	25 participants
		0
Month 6: Self-care (No problem)	Number Analyzed	25 participants
		23
Month 6: Self-care (Slight problem)	Number Analyzed	25 participants
		0
Month 6: Self-care (Moderate problem)	Number Analyzed	25 participants
		1
Month 6: Self-care (Severe problem)	Number Analyzed	25 participants
		1
Month 6: Self-care (Unable to wash or dress)	Number Analyzed	25 participants
		0
Month 9: Self-care (No problem)	Number Analyzed	10 participants
		9
Month 9: Self-care (Slight problem)	Number Analyzed	10 participants
		0
Month 9: Self-care (Moderate problem)	Number Analyzed	10 participants
		0
Month 9: Self-care (Severe problem)	Number Analyzed	10 participants
		1
Month 9: Self-care (Unable to wash or dress)	Number Analyzed	10 participants
		0
Month 12: Self-care (No problem)	Number Analyzed	14 participants
		14
Month 12: Self-care (Slight problem)	Number Analyzed	14 participants
		0
Month 12: Self-care (Moderate problem)	Number Analyzed	14 participants
		0
Month 12: Self-care (Severe problem)	Number Analyzed	14 participants
		0
Month 12: Self-care (Unable to wash or dress)	Number Analyzed	14 participants
		0
Baseline: Usual activities (No problem)	Number Analyzed	51 participants
		24
Baseline: Usual activities (Slight problem)	Number Analyzed	51 participants
		14
Baseline: Usual activities (Moderate problem)	Number Analyzed	51 participants
		9
Baseline: Usual activities (Severe problem)	Number Analyzed	51 participants
		3
Baseline: Usual activities (Unable to do usual activities)	Number Analyzed	51 participants
		1
Day 28: Usual activities (No problem)	Number Analyzed	42 participants
		17
Day 28: Usual activities (Slight problem)	Number Analyzed	42 participants
		13
Day 28: Usual activities (Moderate problem)	Number Analyzed	42 participants
		8
Day 28: Usual activities (Severe problem)	Number Analyzed	42 participants
		3
Day 28: Usual activities (Unable to do usual activities)	Number Analyzed	42 participants
		1
Month 3: Usual activities (No problem)	Number Analyzed	25 participants
		14
Month 3: Usual activities (Slight problem)	Number Analyzed	25 participants
		9
Month 3: Usual activities (Moderate problem)	Number Analyzed	25 participants
		1
Month 3: Usual activities (Severe problem)	Number Analyzed	25 participants
		1
Month 3: Usual activities (Unable to do usual activities)	Number Analyzed	25 participants
		0
Month 6: Usual activities (No problem)	Number Analyzed	25 participants
		17
Month 6: Usual activities (Slight problem)	Number Analyzed	25 participants
		4
Month 6: Usual activities (Moderate problem)	Number Analyzed	25 participants
		2
Month 6: Usual activities (Severe problem)	Number Analyzed	25 participants
		2
Month 6: Usual activities (Unable to do usual activities)	Number Analyzed	25 participants
		0
Month 9: Usual activities (No problem)	Number Analyzed	10 participants
		9
Month 9: Usual activities (Slight problem)	Number Analyzed	10 participants
		0
Month 9: Usual activities (Moderate problem)	Number Analyzed	10 participants
		0
Month 9: Usual activities (Severe problem)	Number Analyzed	10 participants
		0
Month 9: Usual activities (Unable to do usual activities)	Number Analyzed	10 participants
		1
Month 12: Usual activities (No problem)	Number Analyzed	14 participants
		11
Month 12: Usual activities (Slight problem)	Number Analyzed	14 participants
		3
Month 12: Usual activities (Moderate problem)	Number Analyzed	14 participants
		0
Month 12: Usual activities (Severe problem)	Number Analyzed	14 participants
		0
Month 12: Usual activities (Unable to do usual activities)	Number Analyzed	14 participants
		0
Baseline: Pain/Discomfort (No problem)	Number Analyzed	51 participants
		23
Baseline: Pain/Discomfort (Slight problem)	Number Analyzed	51 participants
		16
Baseline: Pain/Discomfort (Moderate problem)	Number Analyzed	51 participants
		12
Baseline: Pain/Discomfort (Severe problem)	Number Analyzed	51 participants
		0
Baseline: Pain/Discomfort (Extreme Pain or discomfort)	Number Analyzed	51 participants
		0
Day 28: Pain/Discomfort (No problem)	Number Analyzed	42 participants
		19
Day 28: Pain/Discomfort (Slight problem)	Number Analyzed	42 participants
		14
Day 28: Pain/Discomfort (Moderate problem)	Number Analyzed	42 participants
		9
Day 28: Pain/Discomfort (Severe problem)	Number Analyzed	42 participants
		0
Day 28: Pain/Discomfort (Extreme Pain or discomfort)	Number Analyzed	42 participants
		0
Month 3: Pain/Discomfort (No problem)	Number Analyzed	26 participants
		11
Month 3: Pain/Discomfort (Slight problem)	Number Analyzed	26 participants
		9
Month 3: Pain/Discomfort (Moderate problem)	Number Analyzed	26 participants
		6
Month 3: Pain/Discomfort (Severe problem)	Number Analyzed	26 participants
		0
Month 3: Pain/Discomfort (Extreme Pain or discomfort)	Number Analyzed	26 participants
		0
Month 6: Pain/Discomfort (No problem)	Number Analyzed	25 participants
		11
Month 6: Pain/Discomfort (Slight problem)	Number Analyzed	25 participants
		5
Month 6: Pain/Discomfort (Moderate problem)	Number Analyzed	25 participants
		7
Month 6: Pain/Discomfort (Severe problem)	Number Analyzed	25 participants
		2
Month 6: Pain/Discomfort (Extreme Pain or discomfort)	Number Analyzed	25 participants
		0
Month 9: Pain/Discomfort (No problem)	Number Analyzed	10 participants
		7
Month 9: Pain/Discomfort (Slight problem)	Number Analyzed	10 participants
		1
Month 9: Pain/Discomfort (Moderate problem)	Number Analyzed	10 participants
		2
Month 9: Pain/Discomfort (Severe problem)	Number Analyzed	10 participants
		0
Month 9: Pain/Discomfort (Extreme Pain or discomfort)	Number Analyzed	10 participants
		0
Month 12: Pain/Discomfort (No problem)	Number Analyzed	14 participants
		7
Month 12: Pain/Discomfort (Slight problem)	Number Analyzed	14 participants
		6
Month 12: Pain/Discomfort (Moderate problem)	Number Analyzed	14 participants
		1
Month 12: Pain/Discomfort (Severe problem)	Number Analyzed	14 participants
		0
Month 12: Pain/Discomfort (Extreme Pain or discomfort)	Number Analyzed	14 participants
		0
Baseline: Anxiety/Depression (No problem)	Number Analyzed	51 participants
		30
Baseline: Anxiety/Depression (Slight problem)	Number Analyzed	51 participants
		12
Baseline: Anxiety/Depression (Moderate problem)	Number Analyzed	51 participants
		7
Baseline: Anxiety/Depression (Severe problem)	Number Analyzed	51 participants
		2
Baseline: Anxiety/Depression (Extreme Anxious or Depressed)	Number Analyzed	51 participants
		0
Day 28: Anxiety/Depression (No problem)	Number Analyzed	42 participants
		28
Day 28: Anxiety/Depression (Slight problem)	Number Analyzed	42 participants
		11
Day 28: Anxiety/Depression (Moderate problem)	Number Analyzed	42 participants
		3
Day 28: Anxiety/Depression (Severe problem)	Number Analyzed	42 participants
		0
Day 28: Anxiety/Depression (Extreme Anxious or Depressed)	Number Analyzed	42 participants
		0
Month 3: Anxiety/Depression (No problem)	Number Analyzed	26 participants
		17
Month 3: Anxiety/Depression (Slight problem)	Number Analyzed	26 participants
		6
Month 3: Anxiety/Depression (Moderate problem)	Number Analyzed	26 participants
		3
Month 3: Anxiety/Depression (Severe problem)	Number Analyzed	26 participants
		0
Month 3: Anxiety/Depression (Extreme Anxious or Depressed)	Number Analyzed	26 participants
		0
Month 6: Anxiety/Depression (No problem)	Number Analyzed	25 participants
		18
Month 6: Anxiety/Depression (Slight problem)	Number Analyzed	25 participants
		4
Month 6: Anxiety/Depression (Moderate problem)	Number Analyzed	25 participants
		3
Month 6: Anxiety/Depression (Severe problem)	Number Analyzed	25 participants
		0
Month 6: Anxiety/Depression (Extreme Anxious or Depressed)	Number Analyzed	25 participants
		0
Month 9: Anxiety/Depression (No problem)	Number Analyzed	10 participants
		9
Month 9: Anxiety/Depression (Slight problem)	Number Analyzed	10 participants
		0
Month 9: Anxiety/Depression (Moderate problem)	Number Analyzed	10 participants
		1
Month 9: Anxiety/Depression (Severe problem)	Number Analyzed	10 participants
		0
Month 9: Anxiety/Depression (Extreme Anxious or Depressed)	Number Analyzed	10 participants
		0
Month 12: Anxiety/Depression (No problem)	Number Analyzed	14 participants
		10
Month 12: Anxiety/Depression (Slight problem)	Number Analyzed	14 participants
		2
Month 12: Anxiety/Depression (Moderate problem)	Number Analyzed	14 participants
		2
Month 12: Anxiety/Depression (Severe problem)	Number Analyzed	14 participants
		0
Month 12: Anxiety/Depression (Extreme Anxious or Depressed)	Number Analyzed	14 participants
		0

18. Secondary Outcome

Title	Phase 2: Change From Baseline Over Time in EQ-5D: Visual Analogue Scale (VAS)
Description	EQ-5D is a self-reported questionnaire used for assessing the overall health status of a participant. The EQ-5D-VAS records the participant's self-rated health on a 20-cm vertical visual analogue scale and is asked to make a global assessment of their current state of health with 0 indicating the worst health they can imagine and 100 indicating the best health they can imagine.
Time Frame	Baseline, Day 28, Month 3, Month 6, Month 9, Month 12

Outcome Measure Data

Analysis Population Description

Participants in Safety Analysis Set with available data were analyzed.

Arm/Group Title		Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description:		Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
Overall Number of Participants Analyzed		51
Mean (Standard Deviation); Unit of Measure: score on a scale
Baseline	Number Analyzed	51 participants
		68.2 (21.8)
Change at Day 28	Number Analyzed	39 participants
		4.1 (14.3)
Change at Month 3	Number Analyzed	26 participants
		9.2 (16.4)
Change at Month 6	Number Analyzed	25 participants
		10.6 (23.6)
Change at Month 9	Number Analyzed	10 participants
		2.2 (25.4)
Change at Month 12	Number Analyzed	14 participants
		16.1 (23.2)

Adverse Events

Time Frame	Adverse Event: First infusion date of brexucabtagene autoleucel to data cutoff date of 09 September 2020 (Maximum duration: 51.7 Months); All-Cause mortality: From enrollment to data cutoff date of 09 September 2020 (Maximum duration: 52.8 Months)
Adverse Event Reporting Description	Adverse Events: The Safety Analysis Set included all enrolled participants treated with any dose of brexucabtagene autoleucel. All-cause mortality: All Enrolled Analysis Set included all enrolled participants in the study.
Arm/Group Title	Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 0.5 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
Arm/Group Description	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 2 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 2 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 0.5 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 0.5 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.	Participants with r/r B-ALL received conditioning chemotherapy (fludarabine 25 mg/m^2 IV over 30 minutes on Day -4, Day -3, and Day -2 and cyclophosphamide 900 mg/m^2 IV over 60 minutes on Day -2) followed by a single infusion of brexucabtagene autoleucel CAR transduced autologous T cells administered IV at a target dose of 1 x 10^6 anti-CD19 CAR T cells/kg of body weight on Day 0. For participants weighing > 100 kg, a maximum flat dose of 1 x 10^8 anti-CD19 CAR T cells/kg of body weight was administered.
All-Cause Mortality
	Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 0.5 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	5/6 (83.33%)	15/28 (53.57%)	15/20 (75.00%)	30/71 (42.25%)
Serious Adverse Events
	Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 0.5 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	6/6 (100.00%)	21/23 (91.30%)	12/16 (75.00%)	41/55 (74.55%)
Blood and lymphatic system disorders
Cytopenia † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Disseminated intravascular coagulation † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	1/55 (1.82%)
Febrile neutropenia † 1	0/6 (0.00%)	0/23 (0.00%)	4/16 (25.00%)	2/55 (3.64%)
Neutropenia † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Pancytopenia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Cardiac disorders
Cardiomyopathy † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Pulseless electrical activity † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Sinus tachycardia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	2/55 (3.64%)
Tachycardia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	3/55 (5.45%)
Gastrointestinal disorders
Colitis † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	1/55 (1.82%)
Diarrhoea † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Gastritis † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Hypoaesthesia oral † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Ileus † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Tongue oedema † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
General disorders
Chills † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Face oedema † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Fatigue † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	2/55 (3.64%)
Multiple organ dysfunction syndrome † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Pyrexia † 1	0/6 (0.00%)	4/23 (17.39%)	1/16 (6.25%)	15/55 (27.27%)
Immune system disorders
Drug hypersensitivity † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Graft versus host disease † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Haemophagocytic lymphohistiocytosis † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	2/55 (3.64%)
Hypogammaglobulinaemia † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Infections and infestations
Bacteraemia † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	1/55 (1.82%)
Catheter bacteraemia † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Cellulitis † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Enterococcal bacteraemia † 1	0/6 (0.00%)	0/23 (0.00%)	2/16 (12.50%)	0/55 (0.00%)
Escherichia bacteraemia † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Escherichia infection † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Escherichia sepsis † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Herpes simplex viraemia † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Influenza † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Osteomyelitis fungal † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Peritonitis † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Pneumocystis jirovecii pneumonia † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Pneumonia † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	3/55 (5.45%)
Pneumonia fungal † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Pneumonia respiratory syncytial viral † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Sepsis † 1	0/6 (0.00%)	4/23 (17.39%)	3/16 (18.75%)	2/55 (3.64%)
Septic shock † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	2/55 (3.64%)
Sinusitis † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Upper respiratory tract infection † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Urinary tract infection † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Injury, poisoning and procedural complications
Brain herniation † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Investigations
Neutrophil count decreased † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Platelet count decreased † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Metabolism and nutrition disorders
Fluid overload † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Tumour lysis syndrome † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Musculoskeletal and connective tissue disorders
Back pain † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	1/55 (1.82%)
Bone pain † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Muscular weakness † 1	2/6 (33.33%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute lymphocytic leukaemia † 1	0/6 (0.00%)	1/23 (4.35%)	4/16 (25.00%)	4/55 (7.27%)
Carcinoma in situ † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Myelodysplastic syndrome † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Nervous system disorders
Aphasia † 1	0/6 (0.00%)	3/23 (13.04%)	1/16 (6.25%)	3/55 (5.45%)
Brain oedema † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Cauda equina syndrome † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Cerebrovascular accident † 1	0/6 (0.00%)	0/23 (0.00%)	2/16 (12.50%)	0/55 (0.00%)
Encephalopathy † 1	2/6 (33.33%)	8/23 (34.78%)	1/16 (6.25%)	4/55 (7.27%)
Facial paralysis † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Headache † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Immune effector cell-associated neurotoxicity syndrome † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Monoplegia † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Paraparesis † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	2/55 (3.64%)
Seizure † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	3/55 (5.45%)
Status epilepticus † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Syncope † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Transient ischaemic attack † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Psychiatric disorders
Confusional state † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	3/55 (5.45%)
Delirium † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Disorientation † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Mental status changes † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Restlessness † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Renal and urinary disorders
Acute kidney injury † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome † 1	0/6 (0.00%)	1/23 (4.35%)	2/16 (12.50%)	0/55 (0.00%)
Acute respiratory failure † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Dyspnoea † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	3/55 (5.45%)
Hypoxia † 1	1/6 (16.67%)	3/23 (13.04%)	2/16 (12.50%)	7/55 (12.73%)
Pleural effusion † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Pulmonary alveolar haemorrhage † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Pulmonary embolism † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Respiratory failure † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	3/55 (5.45%)
Tachypnoea † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Skin and subcutaneous tissue disorders
Rash † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Vascular disorders
Hypertension † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Hypotension † 1	3/6 (50.00%)	9/23 (39.13%)	3/16 (18.75%)	16/55 (29.09%)
Shock † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
1 Term from vocabulary, MedDRA (23.0); † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Phase 1: 2 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 1 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 1: 0.5 x 10^6 Anti-CD19 CAR T Cells/kg	Phase 2: 1 x 10^6 Anti-CD19 CAR T Cells/kg
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	6/6 (100.00%)	23/23 (100.00%)	16/16 (100.00%)	55/55 (100.00%)
Blood and lymphatic system disorders
Anaemia † 1	3/6 (50.00%)	14/23 (60.87%)	4/16 (25.00%)	29/55 (52.73%)
Bone marrow failure † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Bone marrow reticulin fibrosis † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Disseminated intravascular coagulation † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	1/55 (1.82%)
Febrile neutropenia † 1	1/6 (16.67%)	4/23 (17.39%)	2/16 (12.50%)	5/55 (9.09%)
Hypofibrinogenaemia † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	3/55 (5.45%)
Leukocytosis † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	2/55 (3.64%)
Neutropenia † 1	1/6 (16.67%)	7/23 (30.43%)	1/16 (6.25%)	8/55 (14.55%)
Splenic infarction † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Thrombocytopenia † 1	1/6 (16.67%)	3/23 (13.04%)	1/16 (6.25%)	9/55 (16.36%)
Cardiac disorders
Angina pectoris † 1	1/6 (16.67%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Bradycardia † 1	0/6 (0.00%)	0/23 (0.00%)	2/16 (12.50%)	5/55 (9.09%)
Pericardial effusion † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	2/55 (3.64%)
Sinus bradycardia † 1	1/6 (16.67%)	4/23 (17.39%)	0/16 (0.00%)	2/55 (3.64%)
Sinus tachycardia † 1	2/6 (33.33%)	8/23 (34.78%)	2/16 (12.50%)	19/55 (34.55%)
Supraventricular tachycardia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Tachycardia † 1	2/6 (33.33%)	4/23 (17.39%)	6/16 (37.50%)	13/55 (23.64%)
Ventricular tachycardia † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Ear and labyrinth disorders
Tinnitus † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	0/55 (0.00%)
Eye disorders
Dry eye † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	3/55 (5.45%)
Eye pain † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Ocular hyperaemia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Photophobia † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	2/55 (3.64%)
Vision blurred † 1	0/6 (0.00%)	3/23 (13.04%)	2/16 (12.50%)	4/55 (7.27%)
Visual impairment † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Vitreous floaters † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Gastrointestinal disorders
Abdominal distension † 1	1/6 (16.67%)	2/23 (8.70%)	1/16 (6.25%)	4/55 (7.27%)
Abdominal pain † 1	3/6 (50.00%)	5/23 (21.74%)	2/16 (12.50%)	10/55 (18.18%)
Abdominal pain upper † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Anal incontinence † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Ascites † 1	1/6 (16.67%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Constipation † 1	1/6 (16.67%)	11/23 (47.83%)	2/16 (12.50%)	8/55 (14.55%)
Diarrhoea † 1	3/6 (50.00%)	11/23 (47.83%)	6/16 (37.50%)	11/55 (20.00%)
Dry mouth † 1	2/6 (33.33%)	2/23 (8.70%)	1/16 (6.25%)	3/55 (5.45%)
Dyspepsia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	3/55 (5.45%)
Dysphagia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	1/55 (1.82%)
Haematochezia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Haemorrhoids † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	3/55 (5.45%)
Ileus † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Lower gastrointestinal haemorrhage † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Mucous stools † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Nausea † 1	1/6 (16.67%)	11/23 (47.83%)	3/16 (18.75%)	21/55 (38.18%)
Rectal haemorrhage † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Stomatitis † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	1/55 (1.82%)
Toothache † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Vomiting † 1	1/6 (16.67%)	7/23 (30.43%)	2/16 (12.50%)	9/55 (16.36%)
General disorders
Asthenia † 1	0/6 (0.00%)	3/23 (13.04%)	1/16 (6.25%)	3/55 (5.45%)
Catheter site pain † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Chills † 1	3/6 (50.00%)	12/23 (52.17%)	3/16 (18.75%)	18/55 (32.73%)
Face oedema † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	3/55 (5.45%)
Fatigue † 1	1/6 (16.67%)	7/23 (30.43%)	6/16 (37.50%)	13/55 (23.64%)
Feeling abnormal † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Gravitational oedema † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Hypothermia † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Malaise † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	5/55 (9.09%)
Non-cardiac chest pain † 1	0/6 (0.00%)	2/23 (8.70%)	1/16 (6.25%)	2/55 (3.64%)
Oedema † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Oedema peripheral † 1	1/6 (16.67%)	7/23 (30.43%)	4/16 (25.00%)	10/55 (18.18%)
Pain † 1	0/6 (0.00%)	3/23 (13.04%)	0/16 (0.00%)	7/55 (12.73%)
Peripheral swelling † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Pyrexia † 1	6/6 (100.00%)	21/23 (91.30%)	11/16 (68.75%)	49/55 (89.09%)
Hepatobiliary disorders
Hyperbilirubinaemia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	3/55 (5.45%)
Immune system disorders
Drug hypersensitivity † 1	1/6 (16.67%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Hypogammaglobulinaemia † 1	0/6 (0.00%)	3/23 (13.04%)	0/16 (0.00%)	3/55 (5.45%)
Cytokine release syndrome † 1	6/6 (100.00%)	23/23 (100.00%)	13/16 (81.25%)	49/55 (89.09%)
Infections and infestations
Bacteraemia † 1	1/6 (16.67%)	1/23 (4.35%)	1/16 (6.25%)	1/55 (1.82%)
Clostridium difficile infection † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Infection † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Oral candidiasis † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	0/55 (0.00%)
Pneumonia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Skin infection † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Staphylococcal infection † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Upper respiratory tract infection † 1	1/6 (16.67%)	1/23 (4.35%)	0/16 (0.00%)	2/55 (3.64%)
Injury, poisoning and procedural complications
Fall † 1	0/6 (0.00%)	2/23 (8.70%)	2/16 (12.50%)	3/55 (5.45%)
Procedural pain † 1	1/6 (16.67%)	2/23 (8.70%)	0/16 (0.00%)	2/55 (3.64%)
Investigations
Alanine aminotransferase increased † 1	1/6 (16.67%)	8/23 (34.78%)	0/16 (0.00%)	12/55 (21.82%)
Aspartate aminotransferase increased † 1	1/6 (16.67%)	9/23 (39.13%)	0/16 (0.00%)	10/55 (18.18%)
Blood alkaline phosphatase increased † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	4/55 (7.27%)
Blood bilirubin increased † 1	2/6 (33.33%)	4/23 (17.39%)	0/16 (0.00%)	3/55 (5.45%)
Blood creatinine increased † 1	2/6 (33.33%)	0/23 (0.00%)	0/16 (0.00%)	2/55 (3.64%)
Blood fibrinogen decreased † 1	0/6 (0.00%)	4/23 (17.39%)	0/16 (0.00%)	2/55 (3.64%)
C-reactive protein increased † 1	0/6 (0.00%)	0/23 (0.00%)	0/16 (0.00%)	3/55 (5.45%)
Electrocardiogram QT prolonged † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Lymphocyte count decreased † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	4/55 (7.27%)
Neutrophil count decreased † 1	0/6 (0.00%)	8/23 (34.78%)	4/16 (25.00%)	15/55 (27.27%)
Platelet count decreased † 1	1/6 (16.67%)	10/23 (43.48%)	5/16 (31.25%)	18/55 (32.73%)
Transaminases increased † 1	1/6 (16.67%)	1/23 (4.35%)	3/16 (18.75%)	1/55 (1.82%)
Weight decreased † 1	3/6 (50.00%)	3/23 (13.04%)	0/16 (0.00%)	1/55 (1.82%)
Weight increased † 1	2/6 (33.33%)	2/23 (8.70%)	1/16 (6.25%)	0/55 (0.00%)
White blood cell count decreased † 1	0/6 (0.00%)	2/23 (8.70%)	4/16 (25.00%)	14/55 (25.45%)
Metabolism and nutrition disorders
Decreased appetite † 1	0/6 (0.00%)	9/23 (39.13%)	4/16 (25.00%)	8/55 (14.55%)
Dehydration † 1	2/6 (33.33%)	1/23 (4.35%)	1/16 (6.25%)	1/55 (1.82%)
Fluid overload † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Hypercalcaemia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	1/55 (1.82%)
Hyperglycaemia † 1	1/6 (16.67%)	8/23 (34.78%)	5/16 (31.25%)	8/55 (14.55%)
Hyperkalaemia † 1	2/6 (33.33%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Hypermagnesaemia † 1	3/6 (50.00%)	2/23 (8.70%)	0/16 (0.00%)	1/55 (1.82%)
Hypernatraemia † 1	1/6 (16.67%)	1/23 (4.35%)	1/16 (6.25%)	2/55 (3.64%)
Hypoalbuminaemia † 1	3/6 (50.00%)	5/23 (21.74%)	0/16 (0.00%)	1/55 (1.82%)
Hypocalcaemia † 1	2/6 (33.33%)	8/23 (34.78%)	1/16 (6.25%)	9/55 (16.36%)
Hypokalaemia † 1	1/6 (16.67%)	11/23 (47.83%)	2/16 (12.50%)	15/55 (27.27%)
Hypomagnesaemia † 1	1/6 (16.67%)	8/23 (34.78%)	1/16 (6.25%)	12/55 (21.82%)
Hyponatraemia † 1	3/6 (50.00%)	7/23 (30.43%)	2/16 (12.50%)	4/55 (7.27%)
Hypophosphataemia † 1	2/6 (33.33%)	13/23 (56.52%)	3/16 (18.75%)	15/55 (27.27%)
Iron overload † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Metabolic acidosis † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	2/55 (3.64%)
Metabolic alkalosis † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Vitamin D deficiency † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Musculoskeletal and connective tissue disorders
Arthralgia † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	3/55 (5.45%)
Back pain † 1	1/6 (16.67%)	4/23 (17.39%)	0/16 (0.00%)	2/55 (3.64%)
Bone lesion † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Bone pain † 1	1/6 (16.67%)	1/23 (4.35%)	2/16 (12.50%)	3/55 (5.45%)
Coccydynia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Flank pain † 1	2/6 (33.33%)	1/23 (4.35%)	0/16 (0.00%)	0/55 (0.00%)
Muscular weakness † 1	2/6 (33.33%)	5/23 (21.74%)	0/16 (0.00%)	6/55 (10.91%)
Musculoskeletal stiffness † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	0/55 (0.00%)
Myalgia † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	6/55 (10.91%)
Neck pain † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	3/55 (5.45%)
Pain in extremity † 1	1/6 (16.67%)	2/23 (8.70%)	1/16 (6.25%)	2/55 (3.64%)
Nervous system disorders
Aphasia † 1	0/6 (0.00%)	8/23 (34.78%)	2/16 (12.50%)	6/55 (10.91%)
Ataxia † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	0/55 (0.00%)
Cerebral ischaemia † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Cognitive disorder † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	3/55 (5.45%)
Dizziness † 1	1/6 (16.67%)	2/23 (8.70%)	2/16 (12.50%)	8/55 (14.55%)
Dysgeusia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Encephalopathy † 1	4/6 (66.67%)	6/23 (26.09%)	1/16 (6.25%)	9/55 (16.36%)
Headache † 1	1/6 (16.67%)	10/23 (43.48%)	7/16 (43.75%)	20/55 (36.36%)
Lethargy † 1	0/6 (0.00%)	3/23 (13.04%)	2/16 (12.50%)	0/55 (0.00%)
Memory impairment † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Neuropathy peripheral † 1	1/6 (16.67%)	0/23 (0.00%)	2/16 (12.50%)	0/55 (0.00%)
Peripheral sensory neuropathy † 1	1/6 (16.67%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Seizure † 1	1/6 (16.67%)	2/23 (8.70%)	0/16 (0.00%)	0/55 (0.00%)
Somnolence † 1	0/6 (0.00%)	2/23 (8.70%)	1/16 (6.25%)	3/55 (5.45%)
Tremor † 1	1/6 (16.67%)	8/23 (34.78%)	4/16 (25.00%)	15/55 (27.27%)
Psychiatric disorders
Abnormal dreams † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Agitation † 1	0/6 (0.00%)	4/23 (17.39%)	2/16 (12.50%)	7/55 (12.73%)
Anxiety † 1	1/6 (16.67%)	5/23 (21.74%)	2/16 (12.50%)	4/55 (7.27%)
Confusional state † 1	2/6 (33.33%)	6/23 (26.09%)	5/16 (31.25%)	11/55 (20.00%)
Delirium † 1	2/6 (33.33%)	0/23 (0.00%)	2/16 (12.50%)	1/55 (1.82%)
Depression † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	2/55 (3.64%)
Insomnia † 1	1/6 (16.67%)	3/23 (13.04%)	0/16 (0.00%)	7/55 (12.73%)
Mental status changes † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	2/55 (3.64%)
Renal and urinary disorders
Acute kidney injury † 1	1/6 (16.67%)	1/23 (4.35%)	1/16 (6.25%)	1/55 (1.82%)
Dysuria † 1	1/6 (16.67%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Haematuria † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Micturition urgency † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Pollakiuria † 1	0/6 (0.00%)	2/23 (8.70%)	1/16 (6.25%)	0/55 (0.00%)
Urinary incontinence † 1	0/6 (0.00%)	1/23 (4.35%)	2/16 (12.50%)	3/55 (5.45%)
Urinary retention † 1	1/6 (16.67%)	3/23 (13.04%)	2/16 (12.50%)	2/55 (3.64%)
Reproductive system and breast disorders
Gynaecomastia † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	0/55 (0.00%)
Pelvic pain † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Vaginal haemorrhage † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Respiratory, thoracic and mediastinal disorders
Atelectasis † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Cough † 1	1/6 (16.67%)	2/23 (8.70%)	2/16 (12.50%)	7/55 (12.73%)
Dyspnoea † 1	1/6 (16.67%)	3/23 (13.04%)	3/16 (18.75%)	4/55 (7.27%)
Epistaxis † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	3/55 (5.45%)
Hypoxia † 1	2/6 (33.33%)	5/23 (21.74%)	5/16 (31.25%)	12/55 (21.82%)
Nasal congestion † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	3/55 (5.45%)
Oropharyngeal pain † 1	0/6 (0.00%)	4/23 (17.39%)	0/16 (0.00%)	2/55 (3.64%)
Paranasal sinus discomfort † 1	0/6 (0.00%)	0/23 (0.00%)	2/16 (12.50%)	0/55 (0.00%)
Pleural effusion † 1	0/6 (0.00%)	3/23 (13.04%)	2/16 (12.50%)	0/55 (0.00%)
Pneumonitis † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	3/55 (5.45%)
Productive cough † 1	1/6 (16.67%)	0/23 (0.00%)	0/16 (0.00%)	1/55 (1.82%)
Pulmonary oedema † 1	1/6 (16.67%)	2/23 (8.70%)	1/16 (6.25%)	3/55 (5.45%)
Rales † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Rhinitis allergic † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Sinus pain † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Tachypnoea † 1	0/6 (0.00%)	4/23 (17.39%)	0/16 (0.00%)	1/55 (1.82%)
Wheezing † 1	0/6 (0.00%)	2/23 (8.70%)	1/16 (6.25%)	1/55 (1.82%)
Skin and subcutaneous tissue disorders
Alopecia † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Decubitus ulcer † 1	0/6 (0.00%)	2/23 (8.70%)	1/16 (6.25%)	0/55 (0.00%)
Drug eruption † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	0/55 (0.00%)
Dry skin † 1	1/6 (16.67%)	2/23 (8.70%)	1/16 (6.25%)	0/55 (0.00%)
Night sweats † 1	0/6 (0.00%)	1/23 (4.35%)	0/16 (0.00%)	3/55 (5.45%)
Pruritus † 1	0/6 (0.00%)	3/23 (13.04%)	1/16 (6.25%)	3/55 (5.45%)
Rash † 1	0/6 (0.00%)	4/23 (17.39%)	2/16 (12.50%)	5/55 (9.09%)
Rash macular † 1	0/6 (0.00%)	1/23 (4.35%)	1/16 (6.25%)	1/55 (1.82%)
Rash maculo-papular † 1	0/6 (0.00%)	2/23 (8.70%)	0/16 (0.00%)	2/55 (3.64%)
Skin lesion † 1	0/6 (0.00%)	3/23 (13.04%)	0/16 (0.00%)	0/55 (0.00%)
Vascular disorders
Distributive shock † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	1/55 (1.82%)
Flushing † 1	0/6 (0.00%)	3/23 (13.04%)	1/16 (6.25%)	3/55 (5.45%)
Haematoma † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
Hypertension † 1	0/6 (0.00%)	3/23 (13.04%)	2/16 (12.50%)	7/55 (12.73%)
Hypotension † 1	5/6 (83.33%)	13/23 (56.52%)	7/16 (43.75%)	27/55 (49.09%)
Thrombosis † 1	0/6 (0.00%)	0/23 (0.00%)	1/16 (6.25%)	0/55 (0.00%)
1 Term from vocabulary, MedDRA (23.0); † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met:

• The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or
• The study has been completed at all study sites for at least 2 years

Results Point of Contact

• Name/Title: Medical Information
• Organization: Kite, A Gilead Company
• Phone: 844-454-5483(1-844-454-KITE)
• EMail: medinfo@kitepharma.com

Publications of Results:

Oluwole OO, Shah BD, Baer MR, Bishop MR, Holmes H, Schiller GJ, et al. Outcomes of Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia Treated with Prior Blinatumomab in ZUMA-3, a Study of KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy [Abstract S1569]. The 23rd European Hematology Association (EHA) Congress 2018 14-17 June; Stockholm, Sweden.

Sabatino M, Choi K, Chiruvolu V, Better M. Production of Anti-CD19 CAR T Cells for ZUMA-3 and -4: Phase 1/2 Multicenter Studies Evaluating KTE-C19 in Patients With Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia (R/R ALL) [Abstract 711]. Blood 2016;128 (22):1227.

Shah B, Castro J, Gokbuget N, Kersten MJ, Hagenbeek T, Wierda W, et al. ZUMA-3: A Phase 1/2 Multi-center Study Evaluating the Safety and Efficacy of KTE-C19 Anti-CD19 CAR T Cells in Adult Subjects with Relapsed/Refractory B Precursor Acute Lymphoblastic Leukemia (r/r ALL). European Society for Medical Oncology (ESMO) Congress 2016;Abstract 3713.

Shah B, Huynh V, Sender LS, Lee DW, Castro JE, Wierda WG, et al. High Rates of Minimal Residual Disease-Negative (MRD-) Complete Responses (CR) in Adult and Pediatric and Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL) Treated With KTE-C19 (Anti-CD19 Chimeric Antigen Receptor [CAR] T Cells): Preliminary Results of the ZUMA-3 and ZUMA-4 Trials. Blood 2016;128 (22):2803.

Shah B, Stock W, Wierda W, Topp M, Kersten MJ, Houot R, et al. KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T cell Therapy in Adult Patients (Pts) With Relapsed/ Refractory Acute Lymphoblastic Leukemia (R/R ALL) in the ZUMA-3 Trial: Preliminary Results of Novel Safety Interventions [Abstract ALL-025]. Clinical lymphoma, myeloma & leukemia 2017;17:S253.

Shah B, Stock W, Wierda W, Topp MS, Kersten MJ, Houot R, et al. Preliminary Results of Novel Safety Interventions in Adult Patients (Pts) With Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL) in the ZUMA-3 Trial. European Society for Medical Oncology (ESMO) Congress 2017.

Shah B, Wierda WG, Schiller GJ, Bishop MR, Castro JE, Sabatino M, et al. KTE-C19 Chimeric Antigen Receptor (CAR) T Cell Therapy in Adults with High-Burden Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL): Updated Results from Phase 1/2 of ZUMA-3 [Abstract P523]. The 22nd European Hematology Association (EHA) Congress 2017 22-25 June; Madrid, Spain.

Shah BD, Bishop MR, Oluwole OO, Logan A, Baer MR, Donnellan WB, et al. End of Phase I Results of ZUMA-3, A Phase 1/2 Study of KTE-X19, Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy, in Adult Patients (pts) with Relapsed/Refractory (R/R) Acute Lymphoblastic Leukemia (ALL) [Abstract]. J Clin Oncol 2019;37 (15):7006.

Shah BD, Bishop MR, Oluwole OO, Logan AC, Baer MR, Donnellan WB, et al. KTE-X19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia: End of Phase 1 Results of ZUMA-3 [Abstract PS945]. HemaSphere 2019;3 (S1):426.

Shah BD, Oluwole OO, Baer MR, Bishop MR, Holmes H, Schiller GJ, et al. KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy, in Adult Patients with Relapsed/ Refractory Acute Lymphoblastic Leukemia (R/R ALL): Outcomes in Patients Who Were Treated with Prior Blinatumomab in ZUMA-3 [Abstract ALL-128]. Clinical lymphoma, myeloma & leukemia 2018;18 (Supplement 1):S184.

Shah BD, Oluwole OO, Baer MR, Bishop MR, Holmes H, Schiller GJ, et al. Outcomes of Patients Treated With Prior Blinatumomab in ZUMA-3, a Study of KTE-C19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia. ASCO; 2018 01-05 June; Chicago, IL.

Shah BD, Stock W, Wierda WG, Oluwole O, Holmes H, Schiller GJ, et al. Phase 1 Results of ZUMA-3: KTE-C19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL) [Abstract 612]. Blood 2017;130 (Supplement 1):888.

Shah BD, Wierda WG, Schiller GJ, Bishop MR, Castro JE, Sabatino M, et al. Updated results from ZUMA- 3, a phase 1/2 study of KTE-C19 chimeric antigen receptor (CAR) T cell therapy, in adults with high-burden relapsed/refractory acute lymphoblastic leukemia (R/R ALL) [Abstract 3024]. American Society of Clinical Oncology (ASCO) Annual Meeting; 2017 02-06 June; Chicago, Illinois.

Wierda WG, Bishop MR, Oluwole O, Logan AC, Baer MR, Donnellan WB, et al. Updated Phase 1 Results of Zuma-3: Kte-X19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia [Abstract 256]. Biol Blood Marrow Transplant 2019;25 (3):S185.

Wierda WG, Bishop MR, Oluwole OO, Logan AC, Baer MR, Donnellan WB, et al. Updated Phase 1 Results of Zuma-3: Kte-C19, an Anti-CD19 Chimeric Antigen Receptor T Cell Therapy, in Adult Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia [Abstract]. Blood 2018;132 (Supplement 1):897.

Shah BD, Bishop MR, Oluwole OO, Logan AC, Baer MR, Donnellan WB, O'Dwyer KM, Holmes H, Arellano ML, Ghobadi A, Pagel JM, Lin Y, Cassaday RD, Park JH, Abedi M, Castro JE, DeAngelo DJ, Malone AK, Mawad R, Schiller GJ, Rossi JM, Bot A, Shen T, Goyal L, Jain RK, Vezan R, Wierda WG. KTE-X19 anti-CD19 CAR T-cell therapy in adult relapsed/refractory acute lymphoblastic leukemia: ZUMA-3 phase 1 results. Blood. 2021 Jul 8;138(1):11-22. doi: 10.1182/blood.2020009098.

Shah BD, Ghobadi A, Oluwole OO, Logan AC, Boissel N, Cassaday RD, Leguay T, Bishop MR, Topp MS, Tzachanis D, O'Dwyer KM, Arellano ML, Lin Y, Baer MR, Schiller GJ, Park JH, Subklewe M, Abedi M, Minnema MC, Wierda WG, DeAngelo DJ, Stiff P, Jeyakumar D, Feng C, Dong J, Shen T, Milletti F, Rossi JM, Vezan R, Masouleh BK, Houot R. KTE-X19 for relapsed or refractory adult B-cell acute lymphoblastic leukaemia: phase 2 results of the single-arm, open-label, multicentre ZUMA-3 study. Lancet. 2021 Aug 7;398(10299):491-502. doi: 10.1016/S0140-6736(21)01222-8. Epub 2021 Jun 4.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Shah BD, Smith NJ, Feng C, Jeyakumar S, Castaigne JG, Faghmous I, Masouleh BK, Malone DC, Bishop MR. Cost-Effectiveness of KTE-X19 for Adults with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia in the United States. Adv Ther. 2022 Aug;39(8):3678-3695. doi: 10.1007/s12325-022-02201-6. Epub 2022 Jun 21.

• Responsible Party: Gilead Sciences ( Kite, A Gilead Company )
• ClinicalTrials.gov Identifier: NCT02614066
• Other Study ID Numbers: KTE-C19-103; 2015-005009-35 ( EudraCT Number )
• First Submitted: November 23, 2015
• First Posted: November 25, 2015
• Results First Submitted: September 9, 2021
• Results First Posted: November 29, 2021
• Last Update Posted: November 18, 2023