Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients (ELIANA)
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|ClinicalTrials.gov Identifier: NCT02435849|
Recruitment Status : Active, not recruiting
First Posted : May 6, 2015
Results First Posted : November 22, 2021
Last Update Posted : November 22, 2021
|Condition or disease||Intervention/treatment||Phase|
|B-cell Acute Lymphoblastic Leukemia||Biological: CTL019||Phase 2|
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, both of these cohorts have 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||97 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II, Single Arm, Multicenter Trial to Determine the Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell Acute Lymphoblastic Leukemia|
|Actual Study Start Date :||April 8, 2015|
|Actual Primary Completion Date :||January 21, 2020|
|Estimated Study Completion Date :||November 28, 2022|
Experimental: Single dose of CTL019
Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019).
Tisagenlecleucel was administered as a single iv infusion. Dose: 2.0 to 5.0x106 tisagenlecleucel per kg body weight (for patients ≤ 50 kg) or 1.0 to 2.5x108 tisagenlecleucel (for patients >50 kg).
- Percentage of Participants With Overall Remission Rate (ORR) as Determined by IRC Assessment. [ Time Frame: 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 ( IRC) assessment.
- Percentage of Patients Who Achieve Best Overall Response (BOR) or CR or CRi With an MRD Negative Bone Marrow by Central Analysis Using qPCR [ Time Frame: 3 months ]
- Percentage of Patients Who Achieve CR or CRi at Month 6 Without SCT Between CTL019 Infusion and Month 6 Response Assessment. [ Time Frame: 6 months ]
- Duration of Remission (DOR) [ Time Frame: 60 months ]
- Percentage of Patients Who Achieve CR or CRi With Minimal Residual Disease Negative Bone Marrow [ Time Frame: 3 months ]
- Relapse-free Survival [ Time Frame: 60 months ]
- Event-free Survival [ Time Frame: 60 months ]
- Overall Survival [ Time Frame: 60 months ]
- Response at Day 28 +/- 4 Days [ Time Frame: 1 month ]
- Impact of Baseline Tumor Burden on Response [ Time Frame: 60 months ]
- Percentage of Patient Who Achieve CR or CRi and Then Proceed to SCT While in Remission Before Month 6 Response Assessment [ Time Frame: 6 months ]
- Quality of Response Using MRD Disease Assessments Before Treatment at Day 28 +/-4 Days After Treatment Using Central Assessments by qPCR and Before SCT by Local Assessment (Flow or PCR) [ Time Frame: 60 months ]
- Safety of CTL019 Therapy [ Time Frame: 60 months ]
- Characterize in Vivo Cellular PK Profile of CTL019 Cells in Target Tissues [ Time Frame: 60 months ]
- Prevalence and Incidence of Immunogenicity to CTL019 [ Time Frame: 60 months ]
- Effects of CTL019 Therapy on Patient Reported Outcomes [ Time Frame: 60 months ]
- Derivation of a Score to Predict Cytokine Release Syndrome [ Time Frame: 3 months ]
- Describe the Profile of Soluble Immune Factors That May be Key to Cytokine Release Syndrome [ Time Frame: 6 months ]
- Describe Levels of B and T Cells (Blood and Bone Marrow) Prior to and Following CTL019 Infusion for Safety Monitoring [ Time Frame: 3 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02435849
|Study Director:||Novartis Pharmaceuticals||Novartis Pharmaceuticals|