B-cell Acute Lymphoblastic Leukemia, Diffuse Large B-cell Lymphoma
Conditions
Keywords
out of specification, tisagenlecleucel, acute lymphoblastic leukemia, large B-cell lymphoma, relapsed/refractory, pediatric, young adult patients
Brief 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 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.
Detailed description
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
BIOLOGICALCTL019
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 100 Years
Healthy volunteers
No
Inclusion 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,
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants with Adverse Events (AEs) | From Screening up to 3 months for Part 1 and 1 day for Part 2 | Percentage of participants with Serious AEs (SAEs) and non-SAEs |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Overall Remission Rate in Group A (pALL) | Up to 3 months | Overall Remission Rate is defined as the percentage of participants wiho achieve a complete remission (CR) and CR with incomplete blood count recovery (CRi) within 3 months following infusion of CTL019 as determined by the local investigator assessment for participants in group A (part 1). |
| Part 1: Overall Response Rate in Group B (LBCL) | Up to 3 months | ORR is defined as the percentage of patients who achieve a domplete response and partial response within 3 months following infusion of CTL019 as determined by the local investigator assessment for participants in group B (part 1). |
Countries
Canada, Japan
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed