Phase IIIb Study Evaluating the Safety and Effectiveness of Tisagenlecleucel Out of Specification for Commercial Release in Patients Matching Approved Label for B-cell Cancers

What this Trial Is About

Researchers are evaluating the safety of tisagenlecleucel that does not meet standard commercial release specifications in patients with certain blood cancers. This single-arm, open-label Phase IIIb study focuses on pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia pALL and adult patients with relapsed or refractory large B-cell lymphoma LBCL and related lymphomas. The study aims to assess safety in patients treated within the approved label by the Japan Health Authority, with efficacy also evaluated in Part 1 of the study. Participants will receive a single intravenous infusion of CTL019, a biological treatment consisting of CAR-positive viable T cells. The study includes three groups pediatricyoung adult patients with rr pALL, adult patients with rr LBCL, and adult patients with rr non-Hodgkins lymphoma NHL. Each patients batch of tisagenlecleucel that does not meet commercial release specifications will be individually assessed and approved before infusion. After infusion, patients in Part 1 will be followed for three months, while Part 2 patients will be followed for one day. During the study, participants will be monitored for adverse events from screening through the follow-up periods. Researchers will evaluate safety primarily by tracking the percentage of participants experiencing adverse events. In Part 1, key efficacy outcomes such as overall remission and response rates will also be assessed. The study does not include masking or placebo control, and participants will be regularly observed to collect safety and efficacy data throughout the follow-up durations.

Study of Out of Specification for Tisagenlecleucel