All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
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.
B-cell Acute Lymphoblastic Leukemia
Diffuse Large B-cell Lymphoma
Nov 21, 2019
Mar 31, 2022
(Child, Adult, Older Adult)
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Key inclusion criteria:
Signed informed consent/assent must be obtained for this study prior to participation in the study.
Patients for whom the final manufactured tisagenlecleucel product does not meet the commercial release specifications.
Not excluded from commercial manufacturing under the Health Authority-approved tisagenlecleucel prescribing information for their respective country/region.
OOS material has not been deemed to pose an undue safety risk to the patient.
Patient is suffering from a serious or life-threatening disease or condition.
Repeat leukapheresis is not clinically appropriate per the investigator assessment.
Key exclusion criteria:
Patients meeting any of the following criteria are not eligible for inclusion in this study:
Human immunodeficience virus (HIV) positive patients.
Patients with active replication of Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
Patients with primary central nervous system (CNS) lymphoma.
History of hypersensitivity to any drugs or metabolites of similar chemical classes as tisagenlecleucel.
Uncontrolled active infection or inflammation.
Any medical condition identified by the investigator that may impact the assessment of the safety or efficacy outcomes in relation to study treatment.
Pregnant or nursing (lactating) women.