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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-003205-25 | EudraCT Number |
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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.
This was a initially a one cohort, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL. This main cohort completed enrollment. Two new cohorts were added via an amendment, Cohort 1 for high risk B-cell ALL patients at first relapse, and Cohort 2 for feasibility and safety of CTL019 in high risk B-cell ALL in patients that relapsed <6 months post allo-HSCT. Due to lack of recruitment, Cohort 1 and Cohort 2 halted recruitment. This decision was not related to any safety issue.
The study had the following sequential phases: Screening, Pre-Treatment (Cell Product Preparation & Lymphodepleting Chemotherapy), Treatment and Primary Follow-up, Secondary Follow-up (if applicable) and Survival Follow-up. The total duration of the study is 5 years from CTL019 cell infusion.
Efficacy analyses were performed only on the Main Cohort (n=79) who were infused with tisagenlecleucel. However, the data on disposition and demographics presented in this section includes all patients enrolled to the study (98) and all infused patients (80) (Main Cohort + Cohort 1). No patients were enrolled in Cohort 2.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single dose of CTL019 | Experimental | Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTL019 | Biological | Tisagenlecleucel was administered as a single iv infusion. Dose: 2.0 to 5.0x10^6 tisagenlecleucel per kg body weight (for patients ≤ 50 kg) or 1.0 to 2.5x10^8 tisagenlecleucel (for patients >50 kg). |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Overall Remission Rate (ORR) as Determined by Independent Review Committee (IRC) Assessment. | 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 Platelets >100 x 10^9/L and Circulating blasts <1% and No evidence of extramedullary disease, at least 7 days transfusion independency. CRi is defined as all the prior criteria being met, except that the following exists: Neutrophils ≤1.0 x 10^9/L, and/or Platelets ≤100 x 10^9/L, and/or Platelet and/or neutrophil transfusions ≤7 days before the date of the peripheral blood sample for disease assessment. | during the 3 months after tisagenlecleucel administration |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Overall Remission Rate (ORR) as Per IRC From US Manufacturing Facilities in the Main Cohort Only (Key Secondary) | 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. |
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Inclusion Criteria:
Relapsed or refractory pediatric B-cell ALL
Adequate organ function
For relapsed patients, documentation of CD19 tumor expression within 3 months of study entry.
Bone marrow with ≥ 5% lymphoblasts by morphologic assessment at screening.
Life expectancy > 12 weeks.
Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status ≥ 50 at screening
Signed written informed consent and assent forms
Must meet the institutional criteria to undergo leukapheresis or have an acceptable, store leukapheresis product
Must have an apheresis product of non-mobilized cells received and accepted by the manufacturing site.
Cohort 1 only:
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Childrens Hospital Los Angeles SC CTL019 | Los Angeles | California | 90027 | United States | ||
| Stanford Universtiy Medical Center SC - CTL019B2205J - B2206 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36399695 | Derived | Laetsch TW, Maude SL, Rives S, Hiramatsu H, Bittencourt H, Bader P, Baruchel A, Boyer M, De Moerloose B, Qayed M, Buechner J, Pulsipher MA, Myers GD, Stefanski HE, Martin PL, Nemecek E, Peters C, Yanik G, Khaw SL, Davis KL, Krueger J, Balduzzi A, Boissel N, Tiwari R, O'Donovan D, Grupp SA. Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial. J Clin Oncol. 2023 Mar 20;41(9):1664-1669. doi: 10.1200/JCO.22.00642. Epub 2022 Nov 18. | |
| 35422096 |
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Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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This study was conducted in 11 countries with 23 sites.
98 patients met the eligibility criteria and apheresis was accepted by the manufacturing facility, 80 patients were infused in this study: 79 in the Main Cohort and 1 in Cohort 1. No patients were infused in Cohort 2. Patients could discontinue the trial after meeting the eligibility criteria and prior to tisagenlecleucel infusion.
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| ID | Title | Description |
|---|---|---|
| FG000 | Main Cohort: Single Dose of CTL019 | Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019). |
| FG001 | Cohort 1: Single Dose of CTL019 |
| Title | Milestones | Reasons Not Completed | ||||
|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 26, 2020 | May 17, 2023 |
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| 3 months after tisagenlecleucel administration |
| 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) | 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. | 3 months after tisagenlecleucel administration |
| 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) | 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% | 3 months after tisagenlecleucel administration |
| Percentage of Participants Who Achieved CR or CRi Without Hematopoietic Stem Cell Transplantation (HSCT) | These are the participants who achieved CR or CRi without HSCT between tisagenlecleucel (CTL019) infusion and Month 6 response assessment. | 6 months after tisagenlecleucel administration |
| Percentage of Participants Who Achieved CR or CRi and Then Proceeded to Hematopoietic Stem Cell Transplantation (HSCT) While in Remission Prior to Month 6 Resoonse | These are the participants who achieved CR or CRi and then proceeded to HSCT while in remission prior to Month 6 response assessment | 6 months |
| Number of Participants Who Proceeded to Hematopoietic Stem Cell Transplantation (HSCT) After Tisagenlecleucel (CTL019) Infusion | These are the participants who achieved CR or CRi and then proceeded to SCT after being infused by tisagenlecleucel. | up to 6 months |
| Duration of Remission (DOR) | DOR is the time from achievement of CR or CRi at any time post-infusion, whichever occurs first, to relapse or death. | 60 months |
| Site of Involvement of Subsequent Relapse | Anatomical location of relapse in participants who achieved prior CR/CRi subsequent to tisagenlecleucel infusion. | 60 months |
| Relapse-free Survival Per IRC Assessment | 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. | 60 months |
| Event-free Survival Per IRC Assessment | EFS is the time from date of tisagenlecleucel infusion to the earliest of death, relapse or treatment failure. | 60 months |
| Overall Survival (OS) | OS, is the time from date of tisagenlecleucel infusion to the date of death due to any reason. | 60 months |
| Percentage of Participants Attaining CR or CRi at Day 28 +/- 4 Days Post Tisagenlecleucel (CTL019) Infusion by IRC Assessment | These are participants who had a day 28 response (CR or CRi response) by IRC assessment. | 1 month |
| Response as a Function of Baseline Tumor Burden (Tumor Load) in Main Cohort Only | Percentage of participants who achieved BOR of CR or CRi by flow cytometry as a function of baseline bone marrow tumor burden. | 3 months |
| Bone Marrow MRD Status by Flow Cytometry Per IRC Assessment | Percentage of participants who achieved CR or CRi response with bone marrow MRD negative (MRD < 0.01%) after tisagenlecleucel infusion by flow cytometry. | 28 days |
| Tisagenlecleucel Transgene Levels by qPCR in Peripheral Blood by Day 28 Response by Independent Review Committee (IRC) Assessment | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR in peripheral blood. | Month 60 |
| Tisagenlecleucel Transgene Levels by qPCR in Bone Marrow by Day 28 Response by IRC Assessment | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR in bone marrow. | Month 6 |
| Expression of Tisagenlecleucel (CTL019) Detected by Flow Cytometry in Peripheral Blood by Day 28 Disease Response Per IRC Assessment | This is the summary cellular kinetic concentrations for CTL019 by flow cytometry in peripheral blood. It evaluated the persistence of transduced CTL019 cells post-infusion. Observation was up to Month 60 for peripheral blood. | Month 60 |
| Expression of Tisagenlecleucel (CTL019) Detected by Flow Cytometry in Bone Marrow by Day 28 Response by IRC Assessment | This is the summary cellular kinetic concentrations for CTL019 by flow cytometry. It evaluated the persistence of transduced CTL019 cells post-infusion. Observation was up to Month 6 for bone marrow. | Month 6 |
| Pharmacokinetics (PK) Parameter: Cmax by qPCR in Peripheral Blood, by Day 28 Disease Response by IRC | Cmax is the maximum (peak) observed in peripheral blood drug concentration after single dose administration reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas M60 is the actual timepoint for endpoint assessment. The reported Cmax is the summary of maximum level observed based on the data from each patient and based on all the data that's been collected for up to 60 months in a patient. | 60 months |
| Pharmacokinetics (PK) Parameter: Tmax by qPCR in Peripheral Blood, by Day 28 Disease Response by IRC | Tmax is the time to reach maximum (peak) peripheral blood drug concentration after single dose administration (days)", reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas M60 is the actual timepoint for endpoint assessment. The time frame of 60 months refers to the duration for which the data were reviewed to identify the time of Cmax for this measure. | 60 months |
| Pharmacokinetics (PK) Parameter: AUCs by qPCR in Peripheral Blood, by Day 28 Disease Response by IRC | AUC (area under curve) from day of infusion to day 28 or other disease assessment days, in peripheral blood (% or copies/μg x days), reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas 84 days is the actual timepoint for endpoint assessment. : AUC is defined based on the time window i.e., AUC from 0 to 84 days after infusion. Therefore, the time frame specifies the maximum time for up to which the data are used for estimation of AUC (84 days for AUC84d). | 0 to 84 days after infusion |
| Persistence of Tisagenlecleucel (CTL019) in Blood, Bone Marrow and CSF if Available, by qPCR, by Day 28 Response by IRC | Persistence is defined as the time corresponding to last quantifiable transgene level in peripheral blood (Tlast), reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas M60 is the actual timepoint for endpoint assessment. | 60 months |
| Prevalence and Incidence of Immunogenicity to Tisagenlecleucel (CTL019) | This is defined as the percentage of participants who tested positive for anti-mCAR19 antibodies at any time post-baseline, reported by CR/CRi, no response (NR), Unknown and by All participants. . | At any time post-baseline, up to a max. of 60 months |
| Effects of CTL019 Therapy on Patient Reported Outcomes as Measured by PedsQL Questionnaire | The PedsQL questionnaire was for patients ≥ 8-years-old who achieved BOR as CR or CRi within 3 months and the questionnaire was on emotional, social, school, physical, and psychosocial health. Scores are transformed on a scale from 0 to 100, with the sum of all the items over the number of items answered on all the scales. The total scale score is the averaged value of scores of subscales, which means for each subscale and the total scale, the allowed ranges are 0-100. Higher scores on the PedsQL questionnaire for these subscales indicate consistent improvement of health-related quality of life (HRQol). | Month 3, M6, M12, M24, M60 |
| Effects of CTL019 Therapy on Patient Reported Outcomes as Measured by EQ-5D Questionnaire | Results from the EQ-5D questionnaire is for number of participants who achieved CR or CRi at month 60. The EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain & discomfort, anxiety & depression. Respondents are asked to choose the statement in each dimension that best describes their health status on the day surveyed. Their responses are coded as a number (1, 2, or 3) that corresponds to the respective level of severity: 1 indicates no problems, 2 some problems, and 3 severe problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the 5 dimensions. This decision results in a 1-digit number that expresses the level selected for that dimension. The digits for the 5 dimensions can be combined into a 5-digit number that describes the patient's health state. The scores are then normalized to a value from 0-100 where higher scores = better HRQOL & fewer problems or symptoms. | Month 60 |
| Develop a Score Utilizing Clinical and Biomarker Data and Assess Its Ability for Early Prediction of Cytokine Release Syndrome (CRS) | Derivation of a score to predict cytokine release syndrome. Considering the complexity and challenges of building a scoring system based on limited data from the trial, this analysis was not performed. | 3 months |
| Frequent Monitoring of Concentrations of Soluble Immune Factors in Blood (C Reactive Protein & Ferritin) | Profile of soluble immune factors of key inflammatory markers and cytokine parameters in blood by maximum CRS grade that may be key to cytokine release syndrome (CRS). | Maximum post-baseline (approx. 60 months) |
| Frequent Monitoring of Concentrations of Soluble Immune Factors in Blood (All Other Inflammatory Markers) | Profile of soluble immune factors of key inflammatory markers and cytokine parameters in blood by maximum CRS grade that may be key to cytokine release syndrome (CRS). | Maximum post-baseline (approx. 60 months) |
| Percentage Change From Baseline of Levels of B and T Cells (Blood and Bone Marrow) Prior to and Following CTL019 Infusion | Levels of B and T cells (blood and bone marrow) prior to and following CTL019 infusion for safety monitoring | Month 3, Month 12, Maximum post-baseline (approx. 60 months) |
| Percentage of Participants With Overall Remission Rate (ORR) - From Fraunhofer Institute Manufacturing Facility | 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 Fraunhofer Institute manufacturing facility. | 60 months |
| Percentage of Participants With Best Overall Response (BOR) of CR or CRi With Minimal Residue Disease (MRD) Negative Bone Marrow From Fraunhofer Institute Manufacturing Facility as Per IRC | 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 Fraunhofer Institute manufacturing facilities only, by IRC assessment. | 3 months |
| Tisagenlecleucel Transgene Levels by qPCR in Peripheral Blood - Tisagenlecleucel Manufactured From Fraunhofer Institute | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR, by disease response in blood by IRC assessment. The assessment of the efficacy, safety and in vivo cellular pharmacokinetics are for patients infused with tisagenlecleucel manufactured by Fraunhofer Institute. | Month 60 |
| Tisagenlecleucel Transgene Levels by qPCR in Bone Marrow - Tisagenlecleucel Manufactured From Fraunhofer Institute | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR, by disease response in bone marrow by IRC assessment. The assessment of the efficacy, safety and in vivo cellular pharmacokinetics are for patients infused with tisagenlecleucel manufactured by Fraunhofer Institute. | Month 3 |
| Stanford |
| California |
| 94304 |
| United States |
| Children's Healthcare of Atlanta SC CTL019 | Atlanta | Georgia | 30342 | United States |
| University of Michigan . | Ann Arbor | Michigan | 48109-2800 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Children s Mercy Hospital SC - CTL019B2205J | Kansas City | Missouri | 64108 | United States |
| Duke Unversity Medical Center . | Durham | North Carolina | 27705 | United States |
| Oregon Health and Science University Doernbecher Children's Hosp. | Portland | Oregon | 97239-3098 | United States |
| The Childrens Hospital of Philadelphia CHOP | Philadelphia | Pennsylvania | 19104 | United States |
| University of Texas Southwestern Medical Center . | Dallas | Texas | 75235 | United States |
| University of Utah Clinical Trials Office SC - CTL019B2205J | Salt Lake City | Utah | 84108 | United States |
| Novartis Investigative Site | Parkville | Victoria | 3052 | Australia |
| Novartis Investigative Site | Vienna | A 1090 | Austria |
| Novartis Investigative Site | Ghent | 9000 | Belgium |
| Novartis Investigative Site | Toronto | Ontario | M5G 1X8 | Canada |
| Novartis Investigative Site | Montreal | Quebec | H3T 1C5 | Canada |
| Novartis Investigative Site | Paris | Cedex 10 | 75475 | France |
| Novartis Investigative Site | Paris | 75019 | France |
| Novartis Investigative Site | Frankfurt | 60590 | Germany |
| Novartis Investigative Site | Monza | MB | 20900 | Italy |
| Novartis Investigative Site | Kyoto | 606 8507 | Japan |
| Novartis Investigative Site | Oslo | 0424 | Norway |
| Novartis Investigative Site | Esplugues de Llobregat | Barcelona | 08950 | Spain |
| Derived |
| Laetsch TW, Maude SL, Balduzzi A, Rives S, Bittencourt H, Boyer MW, Buechner J, De Moerloose B, Qayed M, Phillips CL, Pulsipher MA, Hiramatsu H, Tiwari R, Grupp SA. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia. Leukemia. 2022 Jun;36(6):1508-1515. doi: 10.1038/s41375-022-01550-z. Epub 2022 Apr 14. |
| 34432863 | Derived | Thudium Mueller K, Grupp SA, Maude SL, Levine JE, Pulsipher MA, Boyer MW, August KJ, Myers GD, Tam CS, Jaeger U, Foley SR, Borchmann P, Schuster SJ, Waller EK, Awasthi R, Potthoff B, Warren A, Waldron ER, McBlane F, Chassot-Agostinho A, Laetsch TW. Tisagenlecleucel immunogenicity in relapsed/refractory acute lymphoblastic leukemia and diffuse large B-cell lymphoma. Blood Adv. 2021 Dec 14;5(23):4980-4991. doi: 10.1182/bloodadvances.2020003844. |
| 34353848 | Derived | Levine JE, Grupp SA, Pulsipher MA, Dietz AC, Rives S, Myers GD, August KJ, Verneris MR, Buechner J, Laetsch TW, Bittencourt H, Baruchel A, Boyer MW, De Moerloose B, Qayed M, Davies SM, Phillips CL, Driscoll TA, Bader P, Schlis K, Wood PA, Mody R, Yi L, Leung M, Eldjerou LK, June CH, Maude SL. Pooled safety analysis of tisagenlecleucel in children and young adults with B cell acute lymphoblastic leukemia. J Immunother Cancer. 2021 Aug;9(8):e002287. doi: 10.1136/jitc-2020-002287. |
| 33496754 | Derived | Buechner J, Grupp SA, Hiramatsu H, Teachey DT, Rives S, Laetsch TW, Yanik GA, Wood P, Awasthi R, Yi L, Chassot-Agostinho A, Eldjerou LK, De Moerloose B. Practical guidelines for monitoring and management of coagulopathy following tisagenlecleucel CAR T-cell therapy. Blood Adv. 2021 Jan 26;5(2):593-601. doi: 10.1182/bloodadvances.2020002757. |
| 31606419 | Derived | Laetsch TW, Myers GD, Baruchel A, Dietz AC, Pulsipher MA, Bittencourt H, Buechner J, De Moerloose B, Davis KL, Nemecek E, Driscoll T, Mechinaud F, Boissel N, Rives S, Bader P, Peters C, Sabnis HS, Grupp SA, Yanik GA, Hiramatsu H, Stefanski HE, Rasouliyan L, Yi L, Shah S, Zhang J, Harris AC. Patient-reported quality of life after tisagenlecleucel infusion in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia: a global, single-arm, phase 2 trial. Lancet Oncol. 2019 Dec;20(12):1710-1718. doi: 10.1016/S1470-2045(19)30493-0. Epub 2019 Oct 9. |
| 29385370 | Derived | Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med. 2018 Feb 1;378(5):439-448. doi: 10.1056/NEJMoa1709866. |
Pediatric B-ALL patients who are very high risk at first relapse.
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| Enrolled and Infused |
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| Discontinued Study Follow-up |
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| COMPLETED | Completed = study follow-up completed |
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| NOT COMPLETED |
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Full Analysis Set (FAS): The Full analysis set comprised all patients who received infusion of tisagenlecleucel.
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| ID | Title | Description |
|---|---|---|
| BG000 | Main Cohort: Single Dose of CTL019 | Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019). |
| BG001 | Cohort 1: Single Dose of CTL019 | Pediatric B-ALL patients who are very high risk at first relapse. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Overall Remission Rate (ORR) as Determined by Independent Review Committee (IRC) Assessment. | 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 Platelets >100 x 10^9/L and Circulating blasts <1% and No evidence of extramedullary disease, at least 7 days transfusion independency. CRi is defined as all the prior criteria being met, except that the following exists: Neutrophils ≤1.0 x 10^9/L, and/or Platelets ≤100 x 10^9/L, and/or Platelet and/or neutrophil transfusions ≤7 days before the date of the peripheral blood sample for disease assessment. | Full Analysis Set (FAS): The Full analysis set comprised all patients who received infusion of tisagenlecleucel in the Main Cohort. | Posted | Number | 95% Confidence Interval | Percentage of participants | during the 3 months after tisagenlecleucel administration |
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| Secondary | Percentage of Participants With Overall Remission Rate (ORR) as Per IRC From US Manufacturing Facilities in the Main Cohort Only (Key Secondary) | 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. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel from US manufacturing facilities. | Posted | Number | 95% Confidence Interval | Percentage of participants | 3 months after tisagenlecleucel administration |
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| Secondary | 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) | 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. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel from US manufacturing facilities. | Posted | Number | 95% Confidence Interval | Percentage of participants | 3 months after tisagenlecleucel administration |
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| Secondary | 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) | 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% | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel from all manufacturing facilities. | Posted | Number | 95% Confidence Interval | Percentage of participants | 3 months after tisagenlecleucel administration |
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| Secondary | Percentage of Participants Who Achieved CR or CRi Without Hematopoietic Stem Cell Transplantation (HSCT) | These are the participants who achieved CR or CRi without HSCT between tisagenlecleucel (CTL019) infusion and Month 6 response assessment. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel. | Posted | Number | 95% Confidence Interval | Percentage of participants | 6 months after tisagenlecleucel administration |
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| Secondary | Percentage of Participants Who Achieved CR or CRi and Then Proceeded to Hematopoietic Stem Cell Transplantation (HSCT) While in Remission Prior to Month 6 Resoonse | These are the participants who achieved CR or CRi and then proceeded to HSCT while in remission prior to Month 6 response assessment | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel. | Posted | Number | 95% Confidence Interval | Percentage of Participants | 6 months |
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| Secondary | Number of Participants Who Proceeded to Hematopoietic Stem Cell Transplantation (HSCT) After Tisagenlecleucel (CTL019) Infusion | These are the participants who achieved CR or CRi and then proceeded to SCT after being infused by tisagenlecleucel. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel. | Posted | Number | Participants | up to 6 months |
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| Secondary | Duration of Remission (DOR) | DOR is the time from achievement of CR or CRi at any time post-infusion, whichever occurs first, to relapse or death. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel and who achieved CR or CRi. | Posted | Median | 95% Confidence Interval | months | 60 months |
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| Secondary | Site of Involvement of Subsequent Relapse | Anatomical location of relapse in participants who achieved prior CR/CRi subsequent to tisagenlecleucel infusion. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel and who achieved CR or CRi. Achieving CR or CRi" requireb confirmation but for this analysis, all patients with one CR/CRi, regardless of confirmation were included. | Posted | Count of Participants | Participants | 60 months |
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| Secondary | Relapse-free Survival Per IRC Assessment | 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. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel. | Posted | Median | 95% Confidence Interval | months | 60 months |
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| Secondary | Event-free Survival Per IRC Assessment | EFS is the time from date of tisagenlecleucel infusion to the earliest of death, relapse or treatment failure. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel | Posted | Median | 95% Confidence Interval | months | 60 months |
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| Secondary | Overall Survival (OS) | OS, is the time from date of tisagenlecleucel infusion to the date of death due to any reason. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel | Posted | Median | 95% Confidence Interval | months | 60 months |
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| Secondary | Percentage of Participants Attaining CR or CRi at Day 28 +/- 4 Days Post Tisagenlecleucel (CTL019) Infusion by IRC Assessment | These are participants who had a day 28 response (CR or CRi response) by IRC assessment. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel. | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 month |
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| Secondary | Response as a Function of Baseline Tumor Burden (Tumor Load) in Main Cohort Only | Percentage of participants who achieved BOR of CR or CRi by flow cytometry as a function of baseline bone marrow tumor burden. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel with a baseline tumor burden assessment. | Posted | Number | 95% Confidence Interval | Percentage of participants | 3 months |
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| Secondary | Bone Marrow MRD Status by Flow Cytometry Per IRC Assessment | Percentage of participants who achieved CR or CRi response with bone marrow MRD negative (MRD < 0.01%) after tisagenlecleucel infusion by flow cytometry. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel | Posted | Number | 95% Confidence Interval | Percentage of participants | 28 days |
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| Secondary | Tisagenlecleucel Transgene Levels by qPCR in Peripheral Blood by Day 28 Response by Independent Review Committee (IRC) Assessment | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR in peripheral blood. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel. Analysis of this particular outcome included a subset of PAS who had evaluation data of the parameter at the specific timepoint. Therefore, no participants in Cohort 1 had at least one blood sample providing evaluable cellular kinetic data for tisagenlecleucel at tis time point, so no data was collected for Cohort 1. | Posted | Geometric Mean | Geometric Coefficient of Variation | copies/ug DNA | Month 60 |
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| Secondary | Tisagenlecleucel Transgene Levels by qPCR in Bone Marrow by Day 28 Response by IRC Assessment | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR in bone marrow. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel parameter under investigation. Analysis of this particular outcome included a subset of PAS who had evaluation data of the parameter at the specific timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | copies/ug DNA | Month 6 |
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| Secondary | Expression of Tisagenlecleucel (CTL019) Detected by Flow Cytometry in Peripheral Blood by Day 28 Disease Response Per IRC Assessment | This is the summary cellular kinetic concentrations for CTL019 by flow cytometry in peripheral blood. It evaluated the persistence of transduced CTL019 cells post-infusion. Observation was up to Month 60 for peripheral blood. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel parameter under investigation. Analysis of this particular outcome included a subset of PAS who had evaluation data of the parameter at the specific timepoint. Therefore, no participants in Cohort 1 had at least one blood sample providing evaluable cellular kinetic data for tisagenlecleucel at tis time point, so no data was collected for Cohort 1. | Posted | Geometric Mean | Geometric Coefficient of Variation | Percentage of CD3+/CTL019+ cells | Month 60 |
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| Secondary | Expression of Tisagenlecleucel (CTL019) Detected by Flow Cytometry in Bone Marrow by Day 28 Response by IRC Assessment | This is the summary cellular kinetic concentrations for CTL019 by flow cytometry. It evaluated the persistence of transduced CTL019 cells post-infusion. Observation was up to Month 6 for bone marrow. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel parameter under investigation. Analysis of this particular outcome included a subset of PAS who had evaluation data of the parameter at the specific timepoint. | Posted | Geometric Mean | Geometric Coefficient of Variation | Percentage of CD3+/CTL019+ cells | Month 6 |
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| Secondary | Pharmacokinetics (PK) Parameter: Cmax by qPCR in Peripheral Blood, by Day 28 Disease Response by IRC | Cmax is the maximum (peak) observed in peripheral blood drug concentration after single dose administration reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas M60 is the actual timepoint for endpoint assessment. The reported Cmax is the summary of maximum level observed based on the data from each patient and based on all the data that's been collected for up to 60 months in a patient. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel. | Posted | Geometric Mean | Geometric Coefficient of Variation | copies/ug | 60 months |
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| Secondary | Pharmacokinetics (PK) Parameter: Tmax by qPCR in Peripheral Blood, by Day 28 Disease Response by IRC | Tmax is the time to reach maximum (peak) peripheral blood drug concentration after single dose administration (days)", reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas M60 is the actual timepoint for endpoint assessment. The time frame of 60 months refers to the duration for which the data were reviewed to identify the time of Cmax for this measure. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel. | Posted | Median | Full Range | days | 60 months |
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| Secondary | Pharmacokinetics (PK) Parameter: AUCs by qPCR in Peripheral Blood, by Day 28 Disease Response by IRC | AUC (area under curve) from day of infusion to day 28 or other disease assessment days, in peripheral blood (% or copies/μg x days), reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas 84 days is the actual timepoint for endpoint assessment. : AUC is defined based on the time window i.e., AUC from 0 to 84 days after infusion. Therefore, the time frame specifies the maximum time for up to which the data are used for estimation of AUC (84 days for AUC84d). | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel. | Posted | Geometric Mean | Geometric Coefficient of Variation | copies/ug*days | 0 to 84 days after infusion |
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| Secondary | Persistence of Tisagenlecleucel (CTL019) in Blood, Bone Marrow and CSF if Available, by qPCR, by Day 28 Response by IRC | Persistence is defined as the time corresponding to last quantifiable transgene level in peripheral blood (Tlast), reported by CR/CRi, no response (NR), Unknown and by All participants. D28 refers to the timepoint for definition of responder populations, whereas M60 is the actual timepoint for endpoint assessment. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel. | Posted | Median | Full Range | days | 60 months |
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| Secondary | Prevalence and Incidence of Immunogenicity to Tisagenlecleucel (CTL019) | This is defined as the percentage of participants who tested positive for anti-mCAR19 antibodies at any time post-baseline, reported by CR/CRi, no response (NR), Unknown and by All participants. . | Safety Set: The Safety set comprised all patients who received tisagenlecleucel infusion in the Main Cohort. No participants in Cohort 1 had immunogenicity data providing evaluable cellular kinetic data for tisagenlecleucel at this time point, so no data was collected for Cohort 1. | Posted | Count of Participants | Participants | No | At any time post-baseline, up to a max. of 60 months |
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| Secondary | Effects of CTL019 Therapy on Patient Reported Outcomes as Measured by PedsQL Questionnaire | The PedsQL questionnaire was for patients ≥ 8-years-old who achieved BOR as CR or CRi within 3 months and the questionnaire was on emotional, social, school, physical, and psychosocial health. Scores are transformed on a scale from 0 to 100, with the sum of all the items over the number of items answered on all the scales. The total scale score is the averaged value of scores of subscales, which means for each subscale and the total scale, the allowed ranges are 0-100. Higher scores on the PedsQL questionnaire for these subscales indicate consistent improvement of health-related quality of life (HRQol). | FAS: the Full analysis set comprised all patients who received infusion of tisagenlecleucel and who had evaluable results at the measured time points. This analysis included a subset of FAS who had any data of the particular questionnaire at the specific timepoint. Therefore, no participants in Cohort 1 had questionnaire data for tisagenlecleucel at the measured time points, so no data was collected for Cohort 1. | Posted | Mean | Standard Error | Scores on a scale | Month 3, M6, M12, M24, M60 |
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| Secondary | Effects of CTL019 Therapy on Patient Reported Outcomes as Measured by EQ-5D Questionnaire | Results from the EQ-5D questionnaire is for number of participants who achieved CR or CRi at month 60. The EQ-5D descriptive system comprises 5 dimensions: mobility, self-care, usual activities, pain & discomfort, anxiety & depression. Respondents are asked to choose the statement in each dimension that best describes their health status on the day surveyed. Their responses are coded as a number (1, 2, or 3) that corresponds to the respective level of severity: 1 indicates no problems, 2 some problems, and 3 severe problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the 5 dimensions. This decision results in a 1-digit number that expresses the level selected for that dimension. The digits for the 5 dimensions can be combined into a 5-digit number that describes the patient's health state. The scores are then normalized to a value from 0-100 where higher scores = better HRQOL & fewer problems or symptoms. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel and who had evaluable results at the measured time points. This analysis included a subset of FAS who had any data of the particular questionnaire at the measured timepoints. Therefore, no participants in Cohort 1 had questionnaire data for tisagenlecleucel at the measured time points, so no data was collected for Cohort 1. | Posted | Number | Participants | Month 60 |
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| Secondary | Develop a Score Utilizing Clinical and Biomarker Data and Assess Its Ability for Early Prediction of Cytokine Release Syndrome (CRS) | Derivation of a score to predict cytokine release syndrome. Considering the complexity and challenges of building a scoring system based on limited data from the trial, this analysis was not performed. | This objective was not associated with any data collection from participants. The original plan was to mathematically derive a score from other endpoints measures (e.g., a score combing age, value of CRP, Ferritin, Interferon gamma, etc.). Those potential biomarkers were used to derive the scores that are already reported in other tables. None of the biomarkers analyzed proved to be prognostic for the onset or severity of CRS, hence finally no score was derived and no data for this objective. | Posted | 3 months |
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| Secondary | Frequent Monitoring of Concentrations of Soluble Immune Factors in Blood (C Reactive Protein & Ferritin) | Profile of soluble immune factors of key inflammatory markers and cytokine parameters in blood by maximum CRS grade that may be key to cytokine release syndrome (CRS). | Safety Set: The Safety set comprised all patients who received tisagenlecleucel infusion with evaluable data. This analysis included a subset of Safety set who had any data of the particular biomarker at the specific timepoint. Therefore, no participants in Cohort 1 had biomarker data at this time point, so no data was collected for Cohort 1. | Posted | Geometric Mean | Geometric Coefficient of Variation | fold-change from baseline | Maximum post-baseline (approx. 60 months) |
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| Secondary | Frequent Monitoring of Concentrations of Soluble Immune Factors in Blood (All Other Inflammatory Markers) | Profile of soluble immune factors of key inflammatory markers and cytokine parameters in blood by maximum CRS grade that may be key to cytokine release syndrome (CRS). | Safety Set: The Safety set comprised all patients who received tisagenlecleucel infusion with evaluable data. This analysis included a subset of Safety set who had any data of the particular biomarker at the specific timepoint. Therefore, no participants in Cohort 1 had biomarker data at this time point, so no data was collected for Cohort 1. | Posted | Geometric Mean | Geometric Coefficient of Variation | fold-change from baseline | Maximum post-baseline (approx. 60 months) |
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| Secondary | Percentage Change From Baseline of Levels of B and T Cells (Blood and Bone Marrow) Prior to and Following CTL019 Infusion | Levels of B and T cells (blood and bone marrow) prior to and following CTL019 infusion for safety monitoring | Safety Set: The Safety set comprised all patients who received tisagenlecleucel infusion. | Posted | Mean | Standard Deviation | percentage change from baseline | Month 3, Month 12, Maximum post-baseline (approx. 60 months) |
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| Secondary | Percentage of Participants With Overall Remission Rate (ORR) - From Fraunhofer Institute Manufacturing Facility | 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 Fraunhofer Institute manufacturing facility. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel from Fraunhofer Institue Manufacturing Facility. Participants in Cohort 1 were from the Fraunhofer Institute manufacturing facility and not the US where the data was collected from, so no data was collected for this Cohort. | Posted | Number | 95% Confidence Interval | Percentage of participants | 60 months |
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| Secondary | Percentage of Participants With Best Overall Response (BOR) of CR or CRi With Minimal Residue Disease (MRD) Negative Bone Marrow From Fraunhofer Institute Manufacturing Facility as Per IRC | 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 Fraunhofer Institute manufacturing facilities only, by IRC assessment. | FAS: The Full analysis set comprised all patients who received infusion of tisagenlecleucel from Fraunhofer Institue Manufacturing Facility. Participants in Cohort 1 were from the Fraunhofer Institute manufacturing facility and not the US where the data was collected from, so no data was collected for this Cohort. | Posted | Number | 95% Confidence Interval | Percentage of participants | 3 months |
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| Secondary | Tisagenlecleucel Transgene Levels by qPCR in Peripheral Blood - Tisagenlecleucel Manufactured From Fraunhofer Institute | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR, by disease response in blood by IRC assessment. The assessment of the efficacy, safety and in vivo cellular pharmacokinetics are for patients infused with tisagenlecleucel manufactured by Fraunhofer Institute. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel from Fraunhofer Institue Manufacturing Facility. Participants in Cohort 1 were from the Fraunhofer Institute manufacturing facility and not the US where the data was collected from, so no data was collected for this Cohort. | Posted | Geometric Least Squares Mean | Geometric Coefficient of Variation | copies/ug | Month 60 |
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| Secondary | Tisagenlecleucel Transgene Levels by qPCR in Bone Marrow - Tisagenlecleucel Manufactured From Fraunhofer Institute | This is the summary of cellular kinetic concentrations for Tisagenlecleucel (CTL019) transgene levels by qPCR, by disease response in bone marrow by IRC assessment. The assessment of the efficacy, safety and in vivo cellular pharmacokinetics are for patients infused with tisagenlecleucel manufactured by Fraunhofer Institute. | PAS: The PAS consisted of patients in the FAS who had at least one sample providing evaluable cellular kinetic data for tisagenlecleucel. Participants in Cohort 1 were from the Fraunhofer Institute manufacturing facility and not the US where the data was collected from, so no data was collected for this Cohort. | Posted | Geometric Least Squares Mean | Geometric Coefficient of Variation | copies/ug | Month 3 |
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| Post-Hoc | All Collected Deaths | On-treatment deaths, which include post-treatment survival follow-up deaths, were collected during the post-infusion period (starting at the day of first infusion) until the end of the study, approx. 60 months. All deaths refers to the sum of on-treatment deaths and post-treatment survival follow-up deaths up to approx. 60 months. | Clinical Database Population: all infused participants in the Main cohort | Posted | Number | Participants | On-treatment deaths: Up to 60 months; Post-treatment survival follow-up deaths: Up to approx. 60 months |
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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.
AE description: Any sign or symptom that occurs during the post-infusion period (starting at the day of first infusion of CTL019 until the end of the study) and safety follow-up. Deaths in the post treatment survival follow-up are not considered Adverse Events while still included in the All-Cause Mortality table.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Main Cohort (On-treatment + Post-treatment Survival Follow-up Deaths) | Single dose of CTL019Edit Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019). | 33 | 79 | 63 | 79 | 79 | 79 |
| EG001 | Cohort 1 (On-treatment + Post-treatment Survival Follow-up Deaths) | Single dose of CTL019Edit Pediatric B-ALL patients who are very high risk at first relapse. | 0 | 1 | 0 | 1 | 1 | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Coagulopathy | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Atrioventricular block first degree | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Left ventricular dysfunction | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Abdominal compartment syndrome | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Neutropenic colitis | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Systemic inflammatory response syndrome | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cholestasis | Hepatobiliary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Allergy to immunoglobulin therapy | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cytokine release syndrome | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Haemophagocytic lymphohistiocytosis | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Candida infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Encephalitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Encephalitis viral | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Enterobacter infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Gastroenteritis Escherichia coli | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Gastroenteritis salmonella | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Human herpesvirus 6 infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Klebsiella infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Mastoiditis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Meningitis bacterial | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Meningitis pneumococcal | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Metapneumovirus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Ophthalmic herpes zoster | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pneumocystis jirovecii pneumonia | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pneumonia respiratory syncytial viral | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Soft tissue infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Staphylococcal abscess | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Staphylococcal bacteraemia | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Varicella zoster virus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Viral haemorrhagic cystitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (25.1) | Systematic Assessment |
| |
| Vasoplegia syndrome | Injury, poisoning and procedural complications | MedDRA (25.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Tumour lysis syndrome | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Bone giant cell tumour benign | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (25.1) | Systematic Assessment |
| |
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (25.1) | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hydrocephalus | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Nervous system disorder | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Renal tubular necrosis | Renal and urinary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Endometriosis | Reproductive system and breast disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Bronchial oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Laryngeal oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Venoocclusive disease | Vascular disorders | MedDRA (25.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Chills | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Face oedema | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Generalised oedema | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cytokine release syndrome | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypogammaglobulinaemia | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood fibrinogen decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood immunoglobulin A decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood immunoglobulin M decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Lymphocyte count decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Serum ferritin increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypervolaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Coagulopathy | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Adrenal insufficiency | Endocrine disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Haemophagocytic lymphohistiocytosis | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Immunodeficiency | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Nail infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (25.1) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (25.1) | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA (25.1) | Systematic Assessment |
| |
| Blood immunoglobulin G decreased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Fibrin D dimer increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA (25.1) | Systematic Assessment |
| |
| Hyperphosphataemia | Metabolism and nutrition disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (25.1) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (25.1) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | novartis.email@novartis.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 28, 2022 | May 17, 2023 | SAP_003.pdf |
| ID | Term |
|---|---|
| D002051 | Burkitt Lymphoma |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| ID | Term |
|---|---|
| D020031 | Epstein-Barr Virus Infections |
| D006566 | Herpesviridae Infections |
| D004266 | DNA Virus Infections |
| D014777 | Virus Diseases |
| D007239 | Infections |
| D014412 | Tumor Virus Infections |
| D016393 | Lymphoma, B-Cell |
| D008228 | Lymphoma, Non-Hodgkin |
| D008223 | Lymphoma |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D007945 | Leukemia, Lymphoid |
| D007938 | Leukemia |
| D006402 | Hematologic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000626284 | tisagenlecleucel |
Not provided
Not provided
Not provided
| Male |
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| Asian |
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| Other |
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| OG001 | Cohort 1: Single Dose of CTL019 | Pediatric B-ALL patients who are very high risk at first relapse. |
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| Participants |
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