Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients (ENSIGN)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02228096 |
Recruitment Status :
Completed
First Posted : August 28, 2014
Results First Posted : November 23, 2020
Last Update Posted : November 23, 2020
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Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Conditions |
B-cell Acute Lymphoblastic Leukemia Relapsed B-cell Acute Lymphoblastic Leukemia Refractory B-cell Acute Lymphoblastic Leukemia |
Intervention |
Biological: CTL019 T-cells |
Enrollment | 75 |
Participant Flow
Recruitment Details | Patients were recruited between 14-Aug-2014 (FPFV) and 22-Sep-2017 (LPFV). At the time of LPLV (24-May-2019), the sponsor had decided to transfer all ongoing patients to a separate long-term follow-up study to complete the planned 15 years of follow-up. These patients are designated with the reason for discontinuation as "terminated by sponsor". |
Pre-assignment Details | "Enrolled" means all eligibility criteria were met and apheresis was accepted by the manufacturing facility. Patients could discontinue the trial after enrollment and prior to tisagenlecleucel infusion. Although 75 patients were enrolled, only 64 were infused as 11 discontinued prior to infusion. |
Arm/Group Title | Tisagenlecleucel (CTL019) - All Participants |
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Pediatric participants with r/r B-cell who were infused with tisagenlecleucel |
Period Title: Overall Study | |
Started | 75 |
Enrolled Into Long-term f/u Protocol | 31 |
Enrolled But Not Infused [1] | 11 |
Enrolled and Infused | 64 |
Completed | 4 [2] |
Not Completed | 71 |
Reason Not Completed | |
Study terminated by Sponsor | 24 |
Lack of Efficacy | 18 |
Death | 12 |
Physician Decision | 3 |
Subject/guardian decision | 2 |
New therapy for study indication | 1 |
Enrolled, not infused | 11 |
[1]
Enrolled means all eligibility criteria were met & apheresis was accepted by manufacturing facility
[2]
Completed = study follow-up completed
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Baseline Characteristics
Arm/Group Title | Tisagenlecleucel (CTL019) - All Participants | Not Infused | Total | |
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Pediatric participants with r/r B-cell who were infused with tisagenlecleucel | Pediatric patients with r/r B-cell who were enrolled in the study but not infused with tisagenlecleucel | Total of all reporting groups | |
Overall Number of Baseline Participants | 64 | 11 | 75 | |
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Enrolled Set: All participants who met all the inclusion/exclusion criteria, and whose apheresis product is received and accepted by the manufacturing facilty
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 64 participants | 11 participants | 75 participants | |
12.4 (5.16) | 15.2 (5.44) | 12.8 (5.26) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 64 participants | 11 participants | 75 participants | |
Female |
34 53.1%
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1 9.1%
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35 46.7%
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Male |
30 46.9%
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10 90.9%
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40 53.3%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
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White | Number Analyzed | 64 participants | 11 participants | 75 participants |
52 | 8 | 60 | ||
Asian | Number Analyzed | 64 participants | 11 participants | 75 participants |
5 | 1 | 6 | ||
Other | Number Analyzed | 64 participants | 11 participants | 75 participants |
7 | 2 | 9 | ||
Weight
Mean (Standard Deviation) Unit of measure: Kg |
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Number Analyzed | 64 participants | 11 participants | 75 participants | |
43.7 (20.10) | 86.2 (41.52) | 47.3 (25.25) | ||
Karnofsky/Lansky performance status
[1] [2] Measure Type: Count of Participants Unit of measure: Participants |
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100 | Number Analyzed | 64 participants | 0 participants | 64 participants |
18 28.1%
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18 28.1%
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90 | Number Analyzed | 64 participants | 0 participants | 64 participants |
28 43.8%
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28 43.8%
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80 | Number Analyzed | 64 participants | 0 participants | 64 participants |
13 20.3%
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13 20.3%
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70 | Number Analyzed | 64 participants | 0 participants | 64 participants |
2 3.1%
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2 3.1%
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60 | Number Analyzed | 64 participants | 0 participants | 64 participants |
1 1.6%
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1 1.6%
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50 | Number Analyzed | 64 participants | 0 participants | 64 participants |
2 3.1%
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2 3.1%
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less than 50 | Number Analyzed | 64 participants | 0 participants | 64 participants |
0 0.0%
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0 0.0%
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[1]
Measure Description: Karnofsky/Lansky performance status helps determine the functional status of a recipient. This status is a critical data field that has been determined to be essential for all outcome-based analyses. Karnofsky Scale: designed for recipients aged 16 years & older; Lansky Scale: designed for recipients 1 year old to less than 16 years old. The Karnofsky/Lansky performances are based from 100 to 0, where 100 is "perfect" health & 0 is death. The scale: Able to carry on normal activity, no special care needed (80- 100); Mild to moderate restriction (50-70); Moderate to severe restriction (10- 40).
[2]
Measure Analysis Population Description: FAS = All subjects who received an infusion of tisagenlecleucel
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Outcome Measures
Adverse Events
Limitations and Caveats
Safety data reported is based on all the 64 infused patients with data cutoff of 24May2019.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
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 the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Results Point of Contact
Name/Title: | Study Director |
Organization: | Novartis Pharmaceuticals |
Phone: | 862-778-8300 |
EMail: | novartis.email@novartis.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
ClinicalTrials.gov Identifier: | NCT02228096 |
Other Study ID Numbers: |
CCTL019B2205J 2015-003736-13 ( EudraCT Number ) |
First Submitted: | August 26, 2014 |
First Posted: | August 28, 2014 |
Results First Submitted: | November 22, 2019 |
Results First Posted: | November 23, 2020 |
Last Update Posted: | November 23, 2020 |