Trial Outcomes & Findings for CART19 to Treat B-Cell Leukemia or Lymphoma That Are Resistant or Refractory to Chemotherapy (NCT NCT01029366)

NCT ID: NCT01029366

Last Updated: 2023-06-22

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

26 participants

Primary outcome timeframe

5 years

Results posted on

2023-06-22

Participant Flow

Study Start/End Dates 17-Mar-2010 to 06-Jul-2015

Participant milestones

Participant milestones
Measure	Chronic Lymphocytic Leukemia (CLL) Subjects Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy. Subjects were given minimum/maximum total dose: 1.5x10⁷/ 5x10⁹.	Acute Lymphocytic Leukemia (ALL) Subjects Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy. Subjects were given minimum/maximum total dose: 1.5x10⁷/ 5x10⁹
Overall Study STARTED	18	8
Overall Study COMPLETED	14	6
Overall Study NOT COMPLETED	4	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Chronic Lymphocytic Leukemia (CLL) Subjects Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy. Subjects were given minimum/maximum total dose: 1.5x10⁷/ 5x10⁹.	Acute Lymphocytic Leukemia (ALL) Subjects Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy. Subjects were given minimum/maximum total dose: 1.5x10⁷/ 5x10⁹
Overall Study Withdrawal by Subject	1	0
Overall Study Death	1	1
Overall Study disease progression	2	0
Overall Study manufacturing failure	0	1

Baseline Characteristics

CART19 to Treat B-Cell Leukemia or Lymphoma That Are Resistant or Refractory to Chemotherapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CLL Subjects n=14 Participants Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy	ALL Subjects n=6 Participants Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy	Total n=20 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=93 Participants	5 Participants n=4 Participants	12 Participants n=27 Participants
Age, Categorical >=65 years	7 Participants n=93 Participants	1 Participants n=4 Participants	8 Participants n=27 Participants
Sex: Female, Male Female	2 Participants n=93 Participants	1 Participants n=4 Participants	3 Participants n=27 Participants
Sex: Female, Male Male	12 Participants n=93 Participants	5 Participants n=4 Participants	17 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants	2 Participants n=4 Participants	2 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=93 Participants	4 Participants n=4 Participants	18 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) White	14 Participants n=93 Participants	5 Participants n=4 Participants	19 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants

PRIMARY outcome

Timeframe: 5 years

Outcome measures

Outcome measures
Measure	All Participants n=20 Participants Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy	ALL Subjects Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy
Number of Participants With Adverse Events	20 participants	—

SECONDARY outcome

Timeframe: 5 years

Efficacy assessments for ALL were performed based on bone marrow and blood morphologic criteria and physical examination findings. The definitions for response are primarily based on the standardized response criteria defined by National Comprehensive Cancer Network (NCCN) Guidelines (NCCN, 2013 v.1). Efficacy assessments for CLL were based on lymphadenopathy, hepatomegaly, splenomegaly, bone marrow and blood morphologic and laboratory assessments. The response criteria are consistent with NCCN Guidelines Version 2.2012 CLL/SLL, which is based on the 2008 International Workshop Group on CLL (IWCLL) revisions of the original guidelines for evaluating disease response released in 1996 by the National Cancer Institute Working Group (NCI/WG).

Outcome measures

Outcome measures
Measure	All Participants n=14 Participants Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy	ALL Subjects n=6 Participants Patients receive CART-19 cells transduced with a lentiviral vector to express anti-CD19 scFv TCRζ:41BB administered on days 0, 1, 2 and 11 in the absence of disease progression or unacceptable toxicity laboratory biomarker analysis polymerase chain reaction reverse transcriptase-polymerase chain reaction anti-CD19-CAR retroviral vector-transduced autologous T cells: Given IV genetically engineered lymphocyte therapy
Overall Response Summary Complete Remission	21.4 percentage of participants	83.4 percentage of participants
Overall Response Summary Partial Remission	21.4 percentage of participants	0 percentage of participants
Overall Response Summary No Remission	57.1 percentage of participants	16.7 percentage of participants
Overall Response Summary Overall Response Rate	42.9 percentage of participants	83.3 percentage of participants

Adverse Events

Chronic Lymphocytic Leukemia

Serious events: 14 serious events

Other events: 14 other events

Deaths: 0 deaths

Acute Lymphocytic Leukemia

Serious events: 6 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Noelle Frey, MD

Abramson Cancer Center of the University of Pennsylvania

Phone: 215-662-6901

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Chronic Lymphocytic Leukemia n=14 participants at risk CART-19 (autologous T cells transduced with CD19 TCR-ζ/4-1BB vector) administered as an IV infusion. Minimum/maximum total dose: 1.5x10\^7 / 5x10\^9.	Acute Lymphocytic Leukemia n=6 participants at risk CART-19 (autologous T cells transduced with CD19 TCR-ζ/4-1BB vector) administered as an IV infusion. Minimum/maximum total dose: 1.5x10\^7 / 5x10\^9.
Immune system disorders Cytokine Release Syndrome	42.9% 6/14 • Number of events 6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	83.3% 5/6 • Number of events 5 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
General disorders pyrexia	28.6% 4/14 • Number of events 4 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	16.7% 1/6 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Blood and lymphatic system disorders Febrile Neutropenia	21.4% 3/14 • Number of events 3 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Blood and lymphatic system disorders anaemia	14.3% 2/14 • Number of events 2 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Pneumonia	14.3% 2/14 • Number of events 2 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	14.3% 2/14 • Number of events 2 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Metabolism and nutrition disorders Tumor Lysis Syndrome	14.3% 2/14 • Number of events 2 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Renal and urinary disorders Acute Kidney Injury	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Cellulitis	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
General disorders Chest Discomfort	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
General disorders Chills	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Clostridium difficle infection	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Psychiatric disorders Confusion State	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Blood and lymphatic system disorders Haemolytic anaemia	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Nervous system disorders Headache	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Investigations Histocytosis Haematophagic	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Vascular disorders Hypertension	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Vascular disorders Hypotension	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Influenza like illness	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Cardiac disorders Myocardial Infarction	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Neutropenic sepsis	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Pneumonia fungal	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
Infections and infestations Pseudomas Infection	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade
General disorders Pain in extremity	7.1% 1/14 • Number of events 1 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade	0.00% 0/6 • 2 years Adverse events during post-infusion period, regardless of study drug relationship, by primary system organ class and maximum CTC grade