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
- Conditions
- Relapsed/Refractory B-precursor Acute Lymphoblastic LeukemiaRelapsed/Refractory B-Cell Non-Hodgkin Lymphoma
- Interventions
- Registration Number
- NCT02625480
- Lead Sponsor
- Kite, A Gilead Company
- Brief Summary
The primary objectives of this study are to evaluate the safety and efficacy of brexucabtagene autoleucel (KTE-X19) in pediatric and adolescent participants with relapsed/refractory (r/r) B-precursor acute lymphoblastic leukemia (ALL) or relapsed or refractory (r/r) B-cell non-Hodgkin lymphoma (NHL).
As of October 2022, no further patients with acute B-cell Acute Lymphoblastic Leukemia (ALL) will be asked to join the study. The study remains open for recruitment for patients that have B-cell Non Hodgkin Lymphoma (NHL).
- Detailed Description
All participants who received KTE-X19, and have completed at least 24 months of protocol assessments, will be transitioned to a separate long-term follow-up (LTFU) study. The purpose of the LTFU study (KT-US-982-5968.) is to complete the remainder of the 15-year follow-up assessments.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 95
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Single Arm Brexucabtagene Autoleucel (KTE-X19) 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 Single Arm Fludarabine 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 Single Arm Cyclophosphamide 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
- Primary Outcome Measures
Name Time Method Phase 2: Overall Complete Remission Rate in the ALL Cohort Up to 24 months Overall complete remission rate will be determined per independent review.
Phase 1: Percentage of Participants Experiencing Adverse Events Defined as Dose-Limiting Toxicities (DLT) 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: Objective Response Rate in the NHL Cohorts Up to 24 months Objective Response Rate will be determined per investigator review.
- Secondary Outcome Measures
Name Time Method CR Rate Within 3 Months Per Independent Review in ALL Cohorts Up to 15 years Minimum Residual Disease Negative Remission Rate in the ALL Cohort Up to 3 months Minimal residual disease (MRD) response rate is defined as MRD \< 10\^-4 per the standard assessment.
Changes Over Time in Patient Reported Outcomes (PRO) Scores in the ALL and NHL Cohorts 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 Survival in the ALL and NHL Cohorts 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.
Allogeneic Stem Cell Transplant Rate in the ALL Cohort Up to 24 months The incidence of allogeneic stem cell transplant will be analyzed.
Overall Complete Remission Rate in the ALL Cohort Up to 15 years Percentage of Participants with Anti-Brexucabtagene Autoleucel (KTE-X19) Antibodies in Blood in the ALL and NHL Cohorts Up to 15 years Relapse-Free Survival for the ALL Cohort 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 Up to 15 years Duration of Remission in the ALL and NHL Cohorts 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 Experiencing Adverse Events and Common Terminology Criteria for Adverse Events (CTCAE) Grade Changes in Safety Laboratory Values in ALL and NHL Cohorts Up to 15 years
Trial Locations
- Locations (30)
Ann & Robert H. Lurie Children's Hospital
🇺🇸Chicago, Illinois, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
University of Miami Hospital & Clinics
🇺🇸Miami, Florida, United States
UCSF Benioff Children's Hospital
🇺🇸San Francisco, California, United States
Monroe-Carell Jr. Children's Hospital at Vanderbilt
🇺🇸Nashville, Tennessee, United States
The Hospital for Sick Children
🇨🇦Toronto, Canada
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Jurasz University Hospital 1; Collegium Medicum
🇵🇱Bydgoszcz, Poland
Wroclaw Medical University
🇵🇱Wroclaw, Poland
Columbia University Irving Medical Center/Morgan Stanley Children's Hospital-NYP
🇺🇸New York, New York, United States
University Medical Center Hamburg-Eppendorf (UKE)
🇩🇪Hamburg, Germany
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Hopital Robert Debre - Sevice d'Hemato-immunologic
🇫🇷Paris Cedex 19, France
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Virginia Health System, Pediatric Hematology/Oncology Clinic
🇺🇸Charlottesville, Virginia, United States
University Hospital Gent
🇧🇪Gent, Belgium
The University of Texas M.D. Anderson Cancer Center
🇺🇸Houston, Texas, United States
University Hospital Brno
🇨🇿Brno, Czechia
Unité d'Oncologie et Hématologie Pédiatriques
🇫🇷Bordeaux, France
Institut d'Hematologie et Oncologie Pediatrique
🇫🇷Lyon, France
Hopital d'Enfants la Timone
🇫🇷Marseille Cedex 5, France
Hospital Sant Joan de Déu
🇪🇸Barcelona, Spain
Bambino Gesù Children's Hospital
🇮🇹Rome, Italy
Prinses Maxima Centrum
🇳🇱Utrecht, Netherlands
Karolinska University Hospital
🇸🇪Stockholm, Sweden
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Kapi'olani Medical Center for Women and Children
🇺🇸Honolulu, Hawaii, United States