Last Update: Jun 25, 2026 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 ClinicalTrials.gov Identifier: [NCT04094311](https://clinicaltrials.gov/ct2/show/NCT04094311) Novartis Reference Number:CCTL019B2302 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") 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. ## 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. Condition B-cell Acute Lymphoblastic Leukemia, Diffuse Large B-cell Lymphoma Phase Phase3 Overall Status Recruiting Number of Participants200 Start Date Nov 21, 2019 Completion Date Mar 31, 2027 Gender All Age(s) 0 Years - 100 Years (Child, Adult, Older Adult) ## Interventions Biological ### CTL019 A single intravenous (i.v.) infusion of CAR-positive viable T cells. ## 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. Japan #### Novartis Investigative Site Recruiting Ishikawa,9208641,Japan #### Novartis Investigative Site Recruiting Fuchū,Tokyo,1838524,Japan #### Novartis Investigative Site Recruiting Setagaya-ku,Tokyo,1578535,Japan #### Novartis Investigative Site Recruiting Shinjuku-ku,Tokyo,1608582,Japan #### Novartis Investigative Site Recruiting Fukuoka,8128582,Japan #### Novartis Investigative Site Recruiting Hiroshima,7348551,Japan #### Novartis Investigative Site Recruiting Niigata,9518520,Japan #### Novartis Investigative Site Recruiting Okayama,7008558,Japan #### Novartis Investigative Site Recruiting Osaka,543-8555,Japan #### Novartis Investigative Site Recruiting Osaka,5458586,Japan #### Novartis Investigative Site Recruiting Suita,Osaka,565-0871,Japan #### Novartis Investigative Site Recruiting Chiba,2608677,Japan #### Novartis Investigative Site Recruiting Bunkyo-ku,Tokyo,1138519,Japan #### Novartis Investigative Site Recruiting Sapporo,Hokkaido,060-8648,Japan #### Novartis Investigative Site Recruiting Nishinomiya,Hyōgo,6638501,Japan #### Novartis Investigative Site Recruiting Izumo,Shimane,6938501,Japan #### Novartis Investigative Site Recruiting Bunkyo Ku,Tokyo,113-8655,Japan #### Novartis Investigative Site Recruiting Chuo Ku,Tokyo,1040045,Japan #### Novartis Investigative Site Recruiting Aomori,0308553,Japan #### Novartis Investigative Site Recruiting Kyoto,6068507,Japan #### Novartis Investigative Site Recruiting Saitama,330-8777,Japan #### Novartis Investigative Site Recruiting Yokohama,Kanagawa,236-0004,Japan #### Novartis Investigative Site Recruiting Kumamoto,Kumamoto,860-8556,Japan #### Novartis Investigative Site Recruiting Bunkyo-ku,Tokyo,113-8603,Japan #### Novartis Investigative Site Recruiting Kita-gun,Kagawa-ken,7610793,Japan #### Novartis Investigative Site Recruiting Wakayama,641-8510,Japan #### Novartis Investigative Site Recruiting Gifu,Gifu,501-1194,Japan #### Novartis Investigative Site Recruiting Kyoto,Kyoto,602-8566,Japan #### Novartis Investigative Site Recruiting Tsu,Mie-ken,514-8507,Japan #### Novartis Investigative Site Recruiting Nagasaki,Nagasaki,852-8501,Japan #### Novartis Investigative Site Recruiting Nagoya,Aichi-ken,4668560,Japan #### Novartis Investigative Site Recruiting Nagoya,Aichi-ken,4678602,Japan #### Novartis Investigative Site Recruiting Tōon,Ehime,7910295,Japan #### Novartis Investigative Site Recruiting Sapporo,Hokkaido,0608543,Japan #### Novartis Investigative Site Recruiting Kobe,Hyōgo,6500047,Japan #### Novartis Investigative Site Recruiting Tsukuba,Ibaraki,3058576,Japan #### Novartis Investigative Site Recruiting Minato-ku,Tokyo,105-8471,Japan #### Novartis Investigative Site Recruiting Sendai,Miyagi,9808574,Japan #### Novartis Investigative Site Recruiting Matsumoto,Nagano,3908621,Japan #### Novartis Investigative Site Recruiting Yufu,Oita Prefecture,8795593,Japan #### Novartis Investigative Site Recruiting Kurashiki,Okayama-ken,7108602,Japan #### Novartis Investigative Site Recruiting Izumi,Osaka,5941101,Japan #### Novartis Investigative Site Recruiting Izumisano,Osaka,5988577,Japan #### Novartis Investigative Site Recruiting Osaka,Osaka,5418567,Japan #### Novartis Investigative Site Recruiting Sakai,Osaka,590-0197,Japan #### Novartis Investigative Site Recruiting Hamamatsu,Shizuoka,4313192,Japan #### Novartis Investigative Site Recruiting Bunkyo Ku,Tokyo,1138431,Japan #### Novartis Investigative Site Recruiting Bunkyo Ku,Tokyo,1138677,Japan ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: #### Novartis Pharmaceuticals Phone: [ +81337978748](tel:+81337978748) Email: