Study Description
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 CTL019 in the patients treated within the approved label by Japan Health Authority in
Part 2. Only for Part 1, in addition to safety, key efficacy of CTL019 will also be
evaluated. This is a single-arm, open-label, multicenter, interventional Phase IIIb study 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 for Part 1 and and r/r ALL and r/r
non-Hodgkin's lymphomas (NHL) for Part 2
Patients whose final manufactured tisagenlecleucel patient-specific batch does not meet
the approved local commercial release specifications are eligible for inclusion. Each
case will be individually assessed and approved by the Novartis manufacturing facility
and the Novartis global medical team (including Patient Safety). Following a single
infusion of CTL019, the patient will be followed for 3 months for Part 1, and 1 day for
Part 2.
Interventions
CTL019
Eligibility Criteria
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:
For part 1, 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. For part 2, exclusion criteria are not set;
however, administration should be performed in accordance with the latest versions
of the package insert of CTL019.
Worldwide Contacts
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