Trial Outcomes & Findings for Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL (NCT NCT03792633)

Last Updated: 2025-06-05

Results Overview

Time from infusion to the first of event or censoring Events = Relapse, No Response, or Death; Censoring =Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

106 participants

Primary outcome timeframe

1 year

Results posted on

2025-06-05

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort A Newly Diagnosed VHR B-ALL or High-Risk Relapse of B	Cohort B Poor Response to Prior B Cell Directed Engineered cell therapy
Overall Study STARTED	55	51
Overall Study COMPLETED	26	21
Overall Study NOT COMPLETED	29	30

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort A Newly Diagnosed VHR B-ALL or High-Risk Relapse of B	Cohort B Poor Response to Prior B Cell Directed Engineered cell therapy
Overall Study Alternative Treatment	17	19
Overall Study Death	2	0
Overall Study Disease progression	8	9
Overall Study Lost to Follow-up	1	1
Overall Study Screen fail	1	0
Overall Study Second malignancy	0	1

Baseline Characteristics

Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort A n=55 Participants Newly Diagnosed VHR B-ALL or High-Risk Relapse of B	Cohort B n=51 Participants Poor Response to Prior B Cell Directed Engineered cell therapy	Total n=106 Participants Total of all reporting groups
Age, Categorical <=18 years	43 Participants n=5 Participants	44 Participants n=7 Participants	87 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=5 Participants	7 Participants n=7 Participants	19 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	12.0 years n=5 Participants	11.6 years n=7 Participants	11.8 years n=5 Participants
Sex: Female, Male Female	20 Participants n=5 Participants	25 Participants n=7 Participants	45 Participants n=5 Participants
Sex: Female, Male Male	35 Participants n=5 Participants	26 Participants n=7 Participants	61 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) White	28 Participants n=5 Participants	40 Participants n=7 Participants	68 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	21 Participants n=5 Participants	7 Participants n=7 Participants	28 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Time from infusion to the first of event or censoring Events = Relapse, No Response, or Death; Censoring =Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.

Outcome measures

Outcome measures
Measure	Cohort A n=52 Participants Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL	Cohort B Patients with poor response to prior B cell directed engineered cell therapy
Number of Subjects With Event-Free Survival in Patients With Newly Diagnosed VHR B-ALL or High-risk Relapse of B-ALL 1 Year After Treatment.	68.9 percentage of subjects Interval 56.8 to 83.5	—

PRIMARY outcome

Timeframe: 1 year

Time from infusion to the first of event or censoring Events = Relapse, No Response (including CR/CRi without B Cell Aplasia), or Death; Censoring =Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.

Outcome measures

Outcome measures
Measure	Cohort A n=48 Participants Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL	Cohort B Patients with poor response to prior B cell directed engineered cell therapy
Number of Subjects With Event-Free Survival in Patients With Poor Response to Prior B Cell Directed Engineered Cell Therapy 1 Year After Treatment.	62 percentage of subject Interval 49.0 to 78.4	—

SECONDARY outcome

Timeframe: 28 Days

Overall remission rate as determined by the response at day 28, computed as the number of subjects with CR or Cri, in patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL.

Outcome measures

Outcome measures
Measure	Cohort A n=52 Participants Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL	Cohort B n=48 Participants Patients with poor response to prior B cell directed engineered cell therapy
Overall Remission Rate	46 Participants	43 Participants

SECONDARY outcome

Timeframe: 28 Days

Population: Only subjects in cohort B analyzed in this measurement

Number of subjects with CR/CRi with B Cell Aplasia at Day 28

Outcome measures

Outcome measures
Measure	Cohort A n=48 Participants Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL	Cohort B Patients with poor response to prior B cell directed engineered cell therapy
Overall Remission Rate (Cohort B)	37 Participants	—

SECONDARY outcome

Timeframe: 1 year

Time from first response of CR/CRi to the first of event or censoring, in responders. Cohort A: Events = Relapse or Death; Censoring = Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first. Cohort B: When defining responders, CR/CRi without B Cell Aplasia is considered no response. Events = Relapse or Death; Censoring = Initiation of New Anticancer Therapy, Last Day of Follow-up/EOS; whichever occurs first.

Outcome measures

Outcome measures
Measure	Cohort A n=52 Participants Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL	Cohort B n=48 Participants Patients with poor response to prior B cell directed engineered cell therapy
Percentage of Subjects With Relapse-free Survival	76.3 percentage of subjects Interval 64.3 to 90.4	80.4 percentage of subjects Interval 67.4 to 95.4

Adverse Events

Cohort A

Serious events: 45 serious events

Other events: 52 other events

Deaths: 2 deaths

Cohort B

Serious events: 32 serious events

Other events: 46 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Regulatory Lead

University of Pennsylvania

Phone: 215-662-4484

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	Cohort A n=52 participants at risk Newly Diagnosed VHR B-ALL or High-Risk Relapse of B	Cohort B n=48 participants at risk Poor Response to Prior B Cell Directed Engineered cell therapy
Blood and lymphatic system disorders Febrile neutropenia	71.2% 37/52 • Number of events 65 • 1 year Adverse events were collected from only those subjects who were given the intervention.	56.2% 27/48 • Number of events 35 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Cardiac disorders Left ventricular systolic dysfunction	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Cardiac disorders Ventricular tachycardia	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Eye disorders Eye disorders - Other (Right soft tissue orbital mass)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Gastrointestinal disorders Colitis	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Gastrointestinal disorders Diarrhea	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Gastrointestinal disorders Dysphagia	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Gastrointestinal disorders Esophagitis	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Gastrointestinal disorders Gastrointestinal disorders - Other (Gastrointestinal hemorrhage)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Gastrointestinal disorders Vomiting	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
General disorders Chills	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
General disorders Fever	28.8% 15/52 • Number of events 18 • 1 year Adverse events were collected from only those subjects who were given the intervention.	18.8% 9/48 • Number of events 10 • 1 year Adverse events were collected from only those subjects who were given the intervention.
General disorders Pain	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Hepatobiliary disorders Hepatic failure	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Immune system disorders Cytokine release syndrome	84.6% 44/52 • Number of events 62 • 1 year Adverse events were collected from only those subjects who were given the intervention.	58.3% 28/48 • Number of events 35 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Catheter related infection	3.8% 2/52 • Number of events 2 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Infections and infestations - Other (Bacterial infectious disorder)	1.9% 1/52 • Number of events 2 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Infections and infestations - Other (Disseminated fungal infection)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Infections and infestations - Other (Metapneumovirus)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Infections and infestations - Other (RSV)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Infections and infestations - Other (S. epidermidis)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Infections and infestations - Other (SARS-CoV-2)	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Lung infection	5.8% 3/52 • Number of events 3 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Myelitis	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Sinusitis	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Skin infection	7.7% 4/52 • Number of events 4 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Upper respiratory infection	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Infections and infestations Wound infection	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Injury, poisoning and procedural complications Vascular access complication	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Investigations Activated partial thromboplastin time prolonged	5.8% 3/52 • Number of events 3 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Investigations Blood bilirubin increased	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Investigations Creatinine increased	5.8% 3/52 • Number of events 3 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Investigations Electrocardiogram QT corrected interval prolonged	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Investigations Fibrinogen decreased	9.6% 5/52 • Number of events 6 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Investigations INR increased	9.6% 5/52 • Number of events 5 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Metabolism and nutrition disorders Dehydration	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Musculoskeletal and connective tissue disorders Bone Pain	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Nervous system disorders Dysphasia	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Nervous system disorders Edema cerebral	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Nervous system disorders Encephalopathy	7.7% 4/52 • Number of events 4 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Nervous system disorders Headache	5.8% 3/52 • Number of events 4 • 1 year Adverse events were collected from only those subjects who were given the intervention.	4.2% 2/48 • Number of events 2 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Pregnancy, puerperium and perinatal conditions Nervous system disorders - Other (CAR Neurotoxicity)	25.0% 13/52 • Number of events 14 • 1 year Adverse events were collected from only those subjects who were given the intervention.	14.6% 7/48 • Number of events 7 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Nervous system disorders Seizure	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	4.2% 2/48 • Number of events 3 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Nervous system disorders Tremor	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Renal and urinary disorders Acute kidney injury	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Respiratory, thoracic and mediastinal disorders Laryngeal edema	3.8% 2/52 • Number of events 2 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Respiratory, thoracic and mediastinal disorders Laryngeal stenosis	1.9% 1/52 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 2 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Respiratory, thoracic and mediastinal disorders Pulmonary edema	7.7% 4/52 • Number of events 4 • 1 year Adverse events were collected from only those subjects who were given the intervention.	0.00% 0/48 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/52 • 1 year Adverse events were collected from only those subjects who were given the intervention.	2.1% 1/48 • Number of events 1 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Vascular disorders Hypotension	19.2% 10/52 • Number of events 10 • 1 year Adverse events were collected from only those subjects who were given the intervention.	6.2% 3/48 • Number of events 3 • 1 year Adverse events were collected from only those subjects who were given the intervention.
Respiratory, thoracic and mediastinal disorders Hypoxia	19.2% 10/52 • Number of events 10 • 1 year Adverse events were collected from only those subjects who were given the intervention.	4.2% 2/48 • Number of events 3 • 1 year Adverse events were collected from only those subjects who were given the intervention.