Study of Out of Specification for Tisagenlecleucel

Official Title

A Phase IIIb Study of the Safety and Efficacy of Tisagenlecleucel Out of Specification for Commercial Release in Patients Who Are Consistent With the Label Indication

Summary:

This study will evaluate the safety of tisagenlecleucel that is out of specification( OOS) for release as commercial product. Specifically, this study will evaluate the safety of tisagenlecleucel in pediatric/young adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (pALL) and adult patients with r/r large B-cell lymphoma (LBCL) including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high-grade B cell lymphoma, and DLBCL arising from follicular lymphoma, that is consistent with the Health Authority-approved indication in the product information for tisagenlecleucel in the respective country/region. In addition to safety, key efficacy of tisagenlecleucel that is out of specification (OOS) for commercial release will also be evaluated.

Trial Description

Primary Outcome:

• Incidence of treatment emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths

Secondary Outcome:

• Group A (pALL): Overall Remission Rate (ORR)
• Group B (LBCL): Overall Response Rate

Patients whose final manufactured tisagenlecleucel batches do not meet the approved local commercial release specifications are eligible for inclusion.Each case will be individually assessed for the expected benefit risk profile prior to release to the patient. Following the infusion of OOS tisagenlecleucel, the patient will be followed for 3 months on this study.

View this trial on ClinicalTrials.gov