Study of Out of Specification for Tisagenlecleucel

June 24, 2026 updated by: Novartis Pharmaceuticals

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
200

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Canada
    • Ontario
      • Hamilton, Ontario, Canada, L8V 5C2
        • Completed
        • Novartis Investigative Site
      • Ottawa, Ontario, Canada, K1H 8L6
        • Withdrawn
        • Novartis Investigative Site
      • Toronto, Ontario, Canada, M5G 1X8
        • Completed
        • Novartis Investigative Site
      • Toronto, Ontario, Canada, M5G 2M9
        • Completed
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, Canada, H1T 2M4
        • Withdrawn
        • Novartis Investigative Site
      • Québec, Quebec, Canada, G1R 2J6
        • Withdrawn
        • Novartis Investigative Site
  • Japan
      • Aomori, Japan, 0308553
        • Recruiting
        • Novartis Investigative Site
      • Chiba, Japan, 2608677
        • Recruiting
        • Novartis Investigative Site
      • Fukuoka, Japan, 8128582
        • Recruiting
        • Novartis Investigative Site
      • Hiroshima, Japan, 7348551
        • Recruiting
        • Novartis Investigative Site
      • Ishikawa, Japan, 9208641
        • Recruiting
        • Novartis Investigative Site
      • Kyoto, Japan, 6068507
        • Recruiting
        • Novartis Investigative Site
      • Niigata, Japan, 9518520
        • Recruiting
        • Novartis Investigative Site
      • Okayama, Japan, 7008558
        • Recruiting
        • Novartis Investigative Site
      • Osaka, Japan, 543-8555
        • Recruiting
        • Novartis Investigative Site
      • Osaka, Japan, 5458586
        • Recruiting
        • Novartis Investigative Site
      • Saitama, Japan, 330-8777
        • Recruiting
        • Novartis Investigative Site
      • Wakayama, Japan, 641-8510
        • Recruiting
        • Novartis Investigative Site
    • Aichi-ken
      • Nagoya, Aichi-ken, Japan, 4678602
        • Recruiting
        • Novartis Investigative Site
      • Nagoya, Aichi-ken, Japan, 4668560
        • Recruiting
        • Novartis Investigative Site
    • Ehime
      • Tōon, Ehime, Japan, 7910295
        • Recruiting
        • Novartis Investigative Site
    • Gifu
      • Gifu, Gifu, Japan, 501-1194
        • Recruiting
        • Novartis Investigative Site
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 0608543
        • Recruiting
        • Novartis Investigative Site
      • Sapporo, Hokkaido, Japan, 060-8648
        • Recruiting
        • Novartis Investigative Site
    • Hyōgo
      • Kobe, Hyōgo, Japan, 6500047
        • Recruiting
        • Novartis Investigative Site
      • Nishinomiya, Hyōgo, Japan, 6638501
        • Recruiting
        • Novartis Investigative Site
    • Ibaraki
      • Tsukuba, Ibaraki, Japan, 3058576
        • Recruiting
        • Novartis Investigative Site
    • Kagawa-ken
      • Kita-gun, Kagawa-ken, Japan, 7610793
        • Recruiting
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama, Kanagawa, Japan, 236-0004
        • Recruiting
        • Novartis Investigative Site
    • Kumamoto
      • Kumamoto, Kumamoto, Japan, 860-8556
        • Recruiting
        • Novartis Investigative Site
    • Kyoto
      • Kyoto, Kyoto, Japan, 602-8566
        • Recruiting
        • Novartis Investigative Site
    • Mie-ken
      • Tsu, Mie-ken, Japan, 514-8507
        • Recruiting
        • Novartis Investigative Site
    • Miyagi
      • Sendai, Miyagi, Japan, 9808574
        • Recruiting
        • Novartis Investigative Site
    • Nagano
      • Matsumoto, Nagano, Japan, 3908621
        • Recruiting
        • Novartis Investigative Site
    • Nagasaki
      • Nagasaki, Nagasaki, Japan, 852-8501
        • Recruiting
        • Novartis Investigative Site
    • Oita Prefecture
      • Yufu, Oita Prefecture, Japan, 8795593
        • Recruiting
        • Novartis Investigative Site
    • Okayama-ken
      • Kurashiki, Okayama-ken, Japan, 7108602
        • Recruiting
        • Novartis Investigative Site
    • Osaka
      • Izumi, Osaka, Japan, 5941101
        • Recruiting
        • Novartis Investigative Site
      • Izumisano, Osaka, Japan, 5988577
        • Recruiting
        • Novartis Investigative Site
      • Osaka, Osaka, Japan, 5418567
        • Recruiting
        • Novartis Investigative Site
      • Sakai, Osaka, Japan, 590-0197
        • Recruiting
        • Novartis Investigative Site
      • Suita, Osaka, Japan, 565-0871
        • Recruiting
        • Novartis Investigative Site
    • Shimane
      • Izumo, Shimane, Japan, 6938501
        • Recruiting
        • Novartis Investigative Site
    • Shizuoka
      • Hamamatsu, Shizuoka, Japan, 4313192
        • Recruiting
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo Ku, Tokyo, Japan, 1138431
        • Recruiting
        • Novartis Investigative Site
      • Bunkyo Ku, Tokyo, Japan, 1138677
        • Recruiting
        • Novartis Investigative Site
      • Bunkyo Ku, Tokyo, Japan, 113-8655
        • Recruiting
        • Novartis Investigative Site
      • Bunkyo-ku, Tokyo, Japan, 113-8603
        • Recruiting
        • Novartis Investigative Site
      • Bunkyo-ku, Tokyo, Japan, 1138519
        • Recruiting
        • Novartis Investigative Site
      • Chuo Ku, Tokyo, Japan, 1040045
        • Recruiting
        • Novartis Investigative Site
      • Fuchū, Tokyo, Japan, 1838524
        • Recruiting
        • Novartis Investigative Site
      • Minato-ku, Tokyo, Japan, 105-8471
        • Recruiting
        • Novartis Investigative Site
      • Setagaya-ku, Tokyo, Japan, 1578535
        • Recruiting
        • Novartis Investigative Site
      • Shinjuku-ku, Tokyo, Japan, 1608582
        • Recruiting
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Biological: CTL019
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Other Names:
  • 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 Names:
  • 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 Names:
  • Tisagenlecleucel

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
From Screening up to 3 months for Part 1 and 1 day for Part 2

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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).
Up to 3 months
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).
Up to 3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 21, 2019

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 31, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 16, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 16, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 18, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 25, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 24, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CCTL019B2302

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings