AALL1721 CTL019G2201J: 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
Study ID: STU-2019-0770
This is a single arm, open-label, multi-center, 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 and 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.
Primary: To evaluate the efficacy of tisagenlecleucel therapy as measured by the 5 year disease-free survival (DFS) by investigator assessment Secondary objectives: (1) To assess the proportion of subjects who are disease free without allogeneic SCT at 1 year (2) To assess overall survival (oS) (3) To assess the proportion of subjects achieving MRD negative CR or CRi at month 3 post-tisagenlecleucel infusion (4) To assess the proportion of subjects in CR or complete remission with incomplete blood count recovery (CRi) with persistent B-cell aplasia over time post tisagenlecleucel infusion (5) To assess the tisagenlecleucel manufacturing success rate in subjects[GreaterThanorequalTo]1 year and [Less Than] 3 years (6) To assess the impact of tisagenlecleucel on health-related Quality of Life (QoL) measures (7) To assess the impact of tisagenlecleucel on neurocognitive measures (8) To assess the safety of tisagenlecleucel therapy (9) To assess the prevalence and incidence of immunogenicity to tisagenlecleucel and its impact on efficacy, safety and cellular kinetics (10) To characterize in vivo cellular kinetic profile (levels, persistence) of tisagenlecleucel transgene and CD3+ CaR-positive viable T cells including second infusion (11) To evaluate the relationship between B-cell and transgene persistence (12) To evaluate dose-exposure-response relationship