Trial Outcomes & Findings for Evaluation of CD19-Specific CAR Engineered Autologous T-Cells for Treatment of Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia (NCT NCT03573700)

Last Updated: 2025-11-18

Results Overview

The primary objectives for the Phase I study portion are to determine the maximum tolerated dose (MTD) and characterize the safety profile and dose-limiting toxicities (DLTs) of treatment with SJCAR19 in pediatric and young adult patient's ≤ 21 years of age, with relapsed or refractory CD19+ ALL. The proportion of participants with dose limiting toxicities are reported.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE1/PHASE2

Target enrollment

24 participants

Primary outcome timeframe

4 weeks post-SJCAR19 infusion

Results posted on

2025-11-18

Participant Flow

This study was posted on http://clinicaltrials.gov. Potential patients were referred from their primary clinical service at St. Jude or through the Physician Referral Office and/or local providers. Twenty-four patients have been enrolled and started treatment since the study opened in July 2018.

Participant milestones

Participant milestones
Measure	Dose Escalation Level 1 (N = 6) Patients assigned to the first dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 1x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Dose Escalation Level 2 (MTD) (N=18 Patients assigned to the second dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 3x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.
Overall Study STARTED	6	18
Overall Study COMPLETED	6	18
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Evaluation of CD19-Specific CAR Engineered Autologous T-Cells for Treatment of Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Dose Escalation Level 1 (N = 6) n=6 Participants Patients assigned to the first dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 1x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Dose Escalation Level 2 (MTD) (N=18) n=18 Participants Patients assigned to the second dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 3x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Total n=24 Participants Total of all reporting groups
Age, Continuous	11.2 years n=202 Participants	4.06 years n=283 Participants	6.07 years n=120 Participants
Sex: Female, Male Female	3 Participants n=202 Participants	10 Participants n=283 Participants	13 Participants n=120 Participants
Sex: Female, Male Male	3 Participants n=202 Participants	8 Participants n=283 Participants	11 Participants n=120 Participants
Race/Ethnicity, Customized White	5 Participants n=202 Participants	15 Participants n=283 Participants	20 Participants n=120 Participants
Race/Ethnicity, Customized Black	1 Participants n=202 Participants	1 Participants n=283 Participants	2 Participants n=120 Participants
Race/Ethnicity, Customized Other	0 Participants n=202 Participants	2 Participants n=283 Participants	2 Participants n=120 Participants

PRIMARY outcome

Timeframe: 4 weeks post-SJCAR19 infusion

Outcome measures

Outcome measures
Measure	Overall (N = 24) n=24 Participants All patients treated on SJCAR19. See above 'Eligibility' for more details.	Dose Escalation Level 1 (N = 6) n=6 Participants Patients assigned to the first dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 1x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Dose Escalation Level 2 (MTD) (N=18) n=18 Participants Patients assigned to the second dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 3x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.
Maximum Tolerated Dose and Dose-limiting Toxicities	2 Participants	1 Participants	1 Participants

PRIMARY outcome

Timeframe: 4 weeks post-SJCAR19 infusion

The primary objective for the Phase II study portion is to evaluate the complete response (CR) rates of SJCAR19 in pediatric and young adult patient's ≤ 21 years of age, with relapsed or refractory CD19+ ALL.

Outcome measures

Outcome measures
Measure	Overall (N = 24) n=24 Participants All patients treated on SJCAR19. See above 'Eligibility' for more details.	Dose Escalation Level 1 (N = 6) n=18 Participants Patients assigned to the first dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 1x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Dose Escalation Level 2 (MTD) (N=18) n=6 Participants Patients assigned to the second dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 3x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.
Complete Response Rate Response: Number of patients with NR (No Response)	4 Participants	3 Participants	1 Participants
Complete Response Rate Response: Number of participants with Complete Response (MRD-positive or negative) status	20 Participants	15 Participants	5 Participants

Adverse Events

Dose Level 1

Serious events: 2 serious events

Other events: 6 other events

Deaths: 3 deaths

Dose Level 2

Serious events: 7 serious events

Other events: 18 other events

Deaths: 9 deaths

Overall

Serious events: 9 serious events

Other events: 24 other events

Deaths: 12 deaths

Serious adverse events

Serious adverse events
Measure	Dose Level 1 n=6 participants at risk Patients assigned to the first dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 1x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Dose Level 2 n=18 participants at risk Patients assigned to the second dose level of CD19 CAR T cells on the protocol dose escalation schema. Patients received lymphodepleting chemotherapy followed by a single infusion of 3x10\^6 CAR+T cells /kg. Dose level changes were based on dose-limiting toxicities. See above 'Eligibility' for more details.	Overall n=24 participants at risk All patients treated on SJCAR19. See above 'Eligibility' for more details.
Immune system disorders Cytokine release syndrome	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	11.1% 2/18 • Number of events 2 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	8.3% 2/24 • Number of events 2 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Blood and lymphatic system disorders Febrile neutropenia	16.7% 1/6 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 2 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	8.3% 2/24 • Number of events 3 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
General disorders Fever	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	11.1% 2/18 • Number of events 2 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	8.3% 2/24 • Number of events 2 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Immune system disorders Immune system disorders - Other	16.7% 1/6 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	8.3% 2/24 • Number of events 2 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Blood and lymphatic system disorders Blood lactate dehydrogenase increased	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	0.00% 0/18 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	0.00% 0/24 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Renal and urinary disorders Acute kidney injury	16.7% 1/6 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	0.00% 0/18 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Infections and infestations Hepatitis viral	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Infections and infestations Infections and infestations - Other, specify	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Metabolism and nutrition disorders Anorexia	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Nervous system disorders Nervous system disorders - Other, specify	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Nervous system disorders Seizure	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/6 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	5.6% 1/18 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.	4.2% 1/24 • Number of events 1 • Adverse events were tracked from on therapy up to 1 year post infusion. In the Phase I study, data on all adverse experiences/toxicities regardless of seriousness were collected. For Phase II (initial and any reinfusions), data on adverse events ≥ grade 3 were recorded. In addition, clinically significant grade 1-2 adverse events related/possibly related to the CAR T-cell product were collected.

Other adverse events

Additional Information

Aimee Talleur, MD

St. Jude Children's Research Hospital

Phone: 8662785833

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place