Under 25, any sex, with B-cell Acute Lymphoblastic Leukemia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Overall Remission Rate (ORR) as Determined by Independent Review Committee (IRC) Assessment.Primary· during the 3 months after tisagenlecleucel administration
Evaluating the efficacy of tisagenlecleucel therapy from all manufacturing facilities as measured by overall remission rate (ORR) during the 3 months after tisagenlecleucel administration. ORR included complete response (CR) and CR with incomplete blood count recovery (CRi) as determined by an Independent Review Committee assessment. Per response criteria defined by National Comprehensive Cancer Network (NCCN), American Society of Hematology (ASH) and International Working Group (IWG) guidelines.
CR is defined as:
Bone marrow \<5% blasts, Peripheral blood: Neutrophils \>1.0 x 10\^9/L, and Pl
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
82.3
72.1 – 90.0
Cohort 1: Single Dose of CTL019
100.0
NA – NA
Percentage of Participants With Overall Remission Rate (ORR) as Per IRC From US Manufacturing Facilities in the Main Cohort Only (Key Secondary)Secondary· 3 months after tisagenlecleucel administration
These are the percentage of participants with ORR who achieved overall remission rate which includes complete response (CR) and CR with incomplete blood count recovery (CRi) as determined by IRC assessment after having been infused with tisagenlecleucel from US manufacturing facilities.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
82.1
70.8 – 90.4
Percentage of Participants With Best Overall Response (BOR) of CR or CRi With Minimal Residue Disease (MRD) Negative Bone Marrow From US Manufacturing Facility as Per IRC in the Main Cohort Only (Key Secondary)Secondary· 3 months after tisagenlecleucel administration
These are the percentage of participants who achieved Best Overall Response (BOR) of complete response (CR) or complete response with incomplete blood count recovery (CRi) with an MRD-negative bone marrow by central analysis using flow cytometry among participants who received tisagenlecleucel from US manufacturing facilities only, by IRC assessment.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
82.1
70.8 – 90.4
Percentage of Participants With Best Overall Response (BOR) of CR or CRi With MRD Negative Bone Marrow by Flow Cytometry From All Manufacturing Facilities as Per IRC in the Main Cohort Only (Key Secondary)Secondary· 3 months after tisagenlecleucel administration
These are the percentage of participants who achieved Best Overall Response (BOR) of CR or CRi with an MRD-negative bone marrow by central analysis using flow cytometry among participants who received tisagenlecleucel from all manufacturing facilities by IRC assessment. MRD negative = MRD% \< 0.01%
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
81.0
70.6 – 89.0
Percentage of Participants Who Achieved CR or CRi Without Hematopoietic Stem Cell Transplantation (HSCT)Secondary· 6 months after tisagenlecleucel administration
These are the participants who achieved CR or CRi without HSCT between tisagenlecleucel (CTL019) infusion and Month 6 response assessment.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
60.8
49.1 – 71.6
Cohort 1: Single Dose of CTL019
100.0
NA – NA
Percentage of Participants Who Achieved CR or CRi and Then Proceeded to Hematopoietic Stem Cell Transplantation (HSCT) While in Remission Prior to Month 6 ResoonseSecondary· 6 months
These are the participants who achieved CR or CRi and then proceeded to HSCT while in remission prior to Month 6 response assessment
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
7.6
2.8 – 15.8
Cohort 1: Single Dose of CTL019
0.0
NA – NA
Number of Participants Who Proceeded to Hematopoietic Stem Cell Transplantation (HSCT) After Tisagenlecleucel (CTL019) InfusionSecondary· up to 6 months
These are the participants who achieved CR or CRi and then proceeded to SCT after being infused by tisagenlecleucel.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
18
Cohort 1: Single Dose of CTL019
0
Duration of Remission (DOR)Secondary· 60 months
DOR is the time from achievement of CR or CRi at any time post-infusion, whichever occurs first, to relapse or death.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
46.8
17.8 – NA
Cohort 1: Single Dose of CTL019
NA
NA – NA
Site of Involvement of Subsequent RelapseSecondary· 60 months
Anatomical location of relapse in participants who achieved prior CR/CRi subsequent to tisagenlecleucel infusion.
BM and/or blood relapse
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
23
Extramedullary only
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
2
Unknown
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
4
Relapse-free Survival Per IRC AssessmentSecondary· 60 months
RFS is the time from achievement of CR or CRi at any time post-infusion, whichever occurs first, to relapse or death due to any cause during CR or CRi.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
46.8
17.8 – NA
Cohort 1: Single Dose of CTL019
NA
NA – NA
Event-free Survival Per IRC AssessmentSecondary· 60 months
EFS is the time from date of tisagenlecleucel infusion to the earliest of death, relapse or treatment failure.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
23.7
9.2 – NA
Cohort 1: Single Dose of CTL019
NA
NA – NA
Overall Survival (OS)Secondary· 60 months
OS, is the time from date of tisagenlecleucel infusion to the date of death due to any reason.
Group
Value
95% CI
Main Cohort: Single Dose of CTL019
NA
45.6 – NA
Cohort 1: Single Dose of CTL019
NA
NA – NA
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse Event (AE) timeframe: AEs were collected during the post-infusion period (starting at the day of 1st infusion until the end of the study), up to maximum duration of 60 months for each patient. Deaths were collected at all points post-treatment (including post-treatment survival follow-up period) until the patient completed the study duration (60 months) or further safety follow-up under the study protocol. Therefore on-treatment deaths include post-treatment survival follow-up deaths..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Main Cohort (On-treatment + Post-treatment Survival Follow-up Deaths)
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04161118 — TisaGenlecleucel in Elderly Patients With First-Relapsed or Primary Refractory Aggressive B-cell Non-Hodgkin Lymphoma
· Phase 2
· terminated
NCT04094311 — Study of Out of Specification for Tisagenlecleucel
· Phase 3
· recruiting
NCT03876769 — Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients
· Phase 2
· active not recruiting
NCT03123939 — Phase III B in Acute Lymphoblastic Leukemia
· Phase 3
· completed
NCT03601442 — CTL019 Out of Specification MAP for ALL or DLBCL Patients
· available
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 13 February 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02435849.