Study Evaluating Brexucabtagene Autoleucel (KTE-X19) in Pediatric and Adolescent Participants With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)
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|ClinicalTrials.gov Identifier: NCT02625480|
Recruitment Status : Recruiting
First Posted : December 9, 2015
Last Update Posted : August 10, 2020
|Condition or disease||Intervention/treatment||Phase|
|Acute Lymphoblastic Leukemia Relapsed Non Hodgkin Lymphoma Refractory Non-Hodgkin Lymphoma||Biological: Brexucabtagene Autoleucel (KTE-X19) Drug: Fludarabine Drug: Cyclophosphamide||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||116 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1/2 Multi-Center Study Evaluating the Safety and Efficacy of KTE-X19 in Pediatric and Adolescent Subjects With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia or Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma (ZUMA-4)|
|Actual Study Start Date :||February 1, 2016|
|Estimated Primary Completion Date :||August 2023|
|Estimated Study Completion Date :||August 2038|
Experimental: Single Arm
A conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by a single infusion of chimeric antigen receptor (CAR) transduced autologous T cells administered intravenously at a target dose of 2 x 10^6 anti-CD19 CAR+ T cells/kg or 1 x 10^6 anti-CD19 CAR+ T cells/kg
Biological: Brexucabtagene Autoleucel (KTE-X19)
- Phase 1: Percentage of Participants Experiencing Adverse Events Defined as Dose-Limiting Toxicities (DLT) [ Time Frame: Up to 28 days ]Dose-limiting toxicity is defined as protocol-defined brexucabtagene autoleucel (KTE-X19)-related events with onset within the first 28 days following brexucabtagene autoleucel (KTE-X19) infusion.
- Phase 2: Overall Complete Remission Rate in the ALL Cohort [ Time Frame: Up to 24 months ]Overall complete remission rate will be determined per independent review.
- Phase 2: Objective Response Rate in the NHL Cohorts [ Time Frame: Up to 24 months ]Objective Response Rate will be determined per investigator review.
- Minimum Residual Disease Negative Remission Rate in the ALL Cohort [ Time Frame: Up to 3 months ]Minimal residual disease (MRD) response rate is defined as MRD < 10^-4 per the standard assessment.
- Allogeneic Stem Cell Transplant Rate in the ALL Cohort [ Time Frame: Up to 24 months ]The incidence of allogeneic stem cell transplant will be analyzed.
- Changes Over Time in Patient Reported Outcomes (PRO) Scores in the ALL and NHL Cohorts [ Time Frame: Up to 15 years ]
The PRO scores will be measured by the Pediatric Quality of Life Inventory (PedsQL) for children and adolescents and European Quality-of-Life-5 Dimension (EQ-5D) for all participants.
The PedsQL comprises of 23 items in the dimensions of physical, emotional, social, and school functioning. Transformed total, physical health summary, and psychosocial health summary scores range from 0-100 with higher scores indicating better health-related quality of life.
The EQ-5D is a generic questionnaire for assessing the participant's overall health status. The EQ-5D consists of a 5 dimension descriptive system including mobility, self-care, usual activities, pain/comfort, and anxiety/depression and a visual analogue scale (EQ-VAS) which allows the respondent to record health. The VAS allows a participant to indicate self-reported health on a vertical scale, ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). The PedsQL scores and EQ-5D scores will be reported.
- Overall Complete Remission Rate in the ALL Cohort [ Time Frame: Up to 15 years ]
- Relapse-Free Survival for the ALL Cohort [ Time Frame: Up to 24 months ]Relapse-Free Survival is defined as the time from the brexucabtagene autoleucel (KTE-X19) infusion date to the date of disease relapse or death from any cause.
- Progression Free Survival in the NHL Cohort [ Time Frame: Up to 15 years ]
- Overall Survival in the ALL and NHL Cohorts [ Time Frame: Up to 15 years ]Overall survival is defined as the time from brexucabtagene autoleucel (KTE-X19) infusion to the date of death from any cause.
- Duration of Remission in the ALL and NHL Cohorts [ Time Frame: Up to 24 months ]Duration of remission is defined as the time between the participant's first complete response per independent review to relapse or any death in the absence of documented relapse.
- Percentage of Participants with Anti-Brexucabtagene Autoleucel (KTE-X19) Antibodies in Blood in the ALL and NHL Cohorts [ Time Frame: Up to 15 years ]
- Percentage of Participants Experiencing Adverse Events and Common Terminology Criteria for Adverse Events (CTCAE) Grade Changes in Safety Laboratory Values in ALL and NHL Cohorts [ Time Frame: Up to 15 years ]
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): NCT02625480
|Contact: Medical Information||1-844-454-5483(1-844-454-KITE)||firstname.lastname@example.org|
|Study Director:||Kite Study Director||Kite, A Gilead Company|