Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

Official Title

A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy

Summary:

This is a single arm, open-label, multi-centre, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment & follow-up, and survival. After tisagenlecleucel infusion, efficacy will be assessed at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Safety will be assessed throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up for lentiviral vector safety will continue under a separate protocol per health authority guidelines.

Trial Description

Primary Outcome:

  • Disease Free Survival (DFS) rate
Secondary Outcome:
  • Percentage of participants who are disease free without allogeneic stem cell transplant (SCT)
  • Overall Survival
  • Percentage of participants achieving MRD negative CR or CRi at Month 3
  • Percentage of participants with in CR or CRi with persistent B-cell aplasia over time post tisagenlecleucel infusion
  • Percentage of participants who have tisagenlecleucel product successfully manufactured over the total number of subjects enrolled for the age ≥1 year and < 3 years
  • Pediatric Quality of Life (PedsQL)
  • European Quality of Life 5 dimensions (EQ-5D-3L & EQ-5D-Y))
  • Impact of tisagenlecleucel on cognitive functioning using Computerized neurocognitive assessment: Detection test
  • Impact of tisagenlecleucel on cognitive functioning usingComputerized neurocognitive assessment: Identification test
  • Impact of tisagenlecleucel on cognitive functioning using Computerized neurocognitive assessment: Groton Maze Learning test
  • Impact of tisagenlecleucel on cognitive functioning using Computerized neurocognitive assessment: One Back test
  • Impact of tisagenlecleucel on cognitive functioning usingComputerized neurocognitive assessment: One card learning test
  • Percentage of participants with pre-existing antibodies
  • Percentage of participants with anti-m CAR19 antibodies post infusion with tisagenlecleucel and % of patients without measureable anti-mCAR19 antibodies
  • Percentage of patients that have measureable anti-mCAR19 antibodies above patient specific cut-point (reported as a %) pre and post tisagenlecleucel infusion categorized by Day 28 response
  • tisagenlecleucel transgene concentration
  • Expression of tisagenlecleucel
  • Persistence of CAR (reported as copies/ug) categorized by the time to B-cell recovery (recovery < 3 months, >3 months to < 6months, > 6 months)
  • Cmax; cellular kinetic parameter of tisagenlecleucel
  • Tmax; cellular kinetic parameter of tisagenlecleucel
  • AUC0-29d and 84d; cellular kinetic parameter of tisagenlecleucel
  • AUC0-Tmax; cellular kinetic parameter of tisagenlecleucel
  • T1/2; cellular kinetic parameter of tisagenlecleucel
  • Clast; cellular kinetic parameter of tisagenlecleucel
  • Tlast; cellular kinetic parameter of tisagenlecleucel
  • Impact of tisagenlecleucel dose on day 29 response
  • AUC 0 - 29d; cellular kinetic parameter of tisagenlecleucel
  • Cmax: cellular kinetic parameter of tisagenlecleucel
  • Tmax: cellular kinetic parameter of tisagenlecleucel
  • T1/2: cellular kinetic parameter of tisagenlecleucel

View this trial on ClinicalTrials.gov

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Resources

Canadian Cancer Society

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