Study of Out of Specification for Tisagenlecleucel
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ClinicalTrials.gov Identifier: NCT04094311 |
Recruitment Status :
Recruiting
First Posted : September 18, 2019
Last Update Posted : October 18, 2022
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Condition or disease | Intervention/treatment | Phase |
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B-cell Acute Lymphoblastic Leukemia Diffuse Large B-cell Lymphoma | Biological: CTL019 | Phase 3 |
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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 42 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | November 21, 2019 |
Estimated Primary Completion Date : | April 14, 2023 |
Estimated Study Completion Date : | April 14, 2023 |

Arm | Intervention/treatment |
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Experimental: Group A: pALL
Pediatric/young adult patients with r/r pALL who meet the indication in the Health Authority-approved CTL019 package insert in the respective country/region whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.
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Biological: CTL019
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Other Name: Tisagenlecleucel |
Experimental: Group B: r/r LBCL
Adult patients with r/r LBCL including 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 package insert for CTL019 in the respective country/region whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.
|
Biological: CTL019
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Other Name: Tisagenlecleucel |
Experimental: Group C: r/r NHL
Adult patients with r/r NHL in consistent with the Health Authority approved indication in the package insert for CTL019 in Japan whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.
|
Biological: CTL019
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Other Name: Tisagenlecleucel |
- Percentage of participants with Adverse Events (AEs) [ Time Frame: 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
- Part 1: Overall Remission Rate in Group A (pALL) [ Time Frame: 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) [ Time Frame: 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).

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04094311
Contact: Novartis Pharmaceuticals | +41613241111 | Novartis.email@novartis.com | |
Contact: Novartis Pharmaceuticals | +81337978748 |
Canada, Ontario | |
Novartis Investigative Site | Completed |
Hamilton, Ontario, Canada, L8V 5C2 | |
Novartis Investigative Site | Withdrawn |
Ottawa, Ontario, Canada, K1H 8L6 | |
Novartis Investigative Site | Completed |
Toronto, Ontario, Canada, M5G 1X8 | |
Novartis Investigative Site | Completed |
Toronto, Ontario, Canada, M5G 2M9 | |
Canada, Quebec | |
Novartis Investigative Site | Withdrawn |
Montreal, Quebec, Canada, H1T 2M4 | |
Canada | |
Novartis Investigative Site | Withdrawn |
Quebec, Canada, G1R 2J6 | |
Japan | |
Novartis Investigative Site | Recruiting |
Nagoya, Aichi, Japan, 466 8560 | |
Novartis Investigative Site | Recruiting |
Fukuoka city, Fukuoka, Japan, 812-8582 | |
Novartis Investigative Site | Recruiting |
Gifu-city, Gifu, Japan, 501-1194 | |
Novartis Investigative Site | Recruiting |
Hiroshima City, Hiroshima, Japan, 734-8551 | |
Novartis Investigative Site | Recruiting |
Sapporo city, Hokkaido, Japan, 060 8648 | |
Novartis Investigative Site | Recruiting |
Kobe-city, Hyogo, Japan, 650-0047 | |
Novartis Investigative Site | Recruiting |
Nishinomiya, Hyogo, Japan, 663 8501 | |
Novartis Investigative Site | Recruiting |
Tsukuba city, Ibaraki, Japan, 305-8576 | |
Novartis Investigative Site | Recruiting |
Kanazawa-city, Ishikawa, Japan, 920-8641 | |
Novartis Investigative Site | Recruiting |
Kita-gun, Kagawa, Japan, 761-0793 | |
Novartis Investigative Site | Recruiting |
Yokohama-city, Kanagawa, Japan, 236-0004 | |
Novartis Investigative Site | Recruiting |
Kyoto-city, Kyoto, Japan, 602-8566 | |
Novartis Investigative Site | Recruiting |
Tsu-city, Mie, Japan, 514-8507 | |
Novartis Investigative Site | Recruiting |
Sendai city, Miyagi, Japan, 980 8574 | |
Novartis Investigative Site | Recruiting |
Matsumoto-city, Nagano, Japan, 390-8621 | |
Novartis Investigative Site | Recruiting |
Nagasaki-city, Nagasaki, Japan, 852-8501 | |
Novartis Investigative Site | Recruiting |
Kurashiki-city, Okayama, Japan, 710-8602 | |
Novartis Investigative Site | Recruiting |
Okayama-city, Okayama, Japan, 700-8558 | |
Novartis Investigative Site | Recruiting |
Osaka Sayama, Osaka, Japan, 589 8511 | |
Novartis Investigative Site | Recruiting |
Osaka-city, Osaka, Japan, 541-8567 | |
Novartis Investigative Site | Recruiting |
Osaka-city, Osaka, Japan, 543-8555 | |
Novartis Investigative Site | Recruiting |
Suita city, Osaka, Japan, 565 0871 | |
Novartis Investigative Site | Recruiting |
Hamamatsu-city, Shizuoka, Japan, 431-3192 | |
Novartis Investigative Site | Recruiting |
Bunkyo ku, Tokyo, Japan, 113 8655 | |
Novartis Investigative Site | Recruiting |
Bunkyo ku, Tokyo, Japan, 113-8431 | |
Novartis Investigative Site | Recruiting |
Bunkyo ku, Tokyo, Japan, 113-8677 | |
Novartis Investigative Site | Recruiting |
Bunkyo-ku, Tokyo, Japan, 113-8519 | |
Novartis Investigative Site | Recruiting |
Chuo ku, Tokyo, Japan, 104 0045 | |
Novartis Investigative Site | Recruiting |
Minato-ku, Tokyo, Japan, 105-8471 | |
Novartis Investigative Site | Recruiting |
Setagaya-ku, Tokyo, Japan, 157-8535 | |
Novartis Investigative Site | Recruiting |
Shinjuku-ku, Tokyo, Japan, 160 8582 | |
Novartis Investigative Site | Recruiting |
Chiba, Japan, 260 8677 | |
Novartis Investigative Site | Recruiting |
Kyoto, Japan, 606 8507 | |
Novartis Investigative Site | Recruiting |
Niigata, Japan, 951 8520 | |
Novartis Investigative Site | Recruiting |
Osaka, Japan, 545-8586 | |
Novartis Investigative Site | Recruiting |
Saitama, Japan, 330 8777 |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT04094311 |
Other Study ID Numbers: |
CCTL019B2302 |
First Posted: | September 18, 2019 Key Record Dates |
Last Update Posted: | October 18, 2022 |
Last Verified: | October 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
out of specification tisagenlecleucel acute lymphoblastic leukemia large B-cell lymphoma |
relapsed/refractory pediatric young adult patients |
Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Lymphoid Lymphoma, B-Cell Lymphoma, Large B-Cell, Diffuse Lymphoma Neoplasms by Histologic Type Neoplasms |
Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Leukemia Lymphoma, Non-Hodgkin |