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B-cell Acute Lymphoblastic LeukemiaPhase 3NCT04094311

Study of Out of Specification for Tisagenlecleucel

A Phase 3 study testing CTL019 in people with B-cell Acute Lymphoblastic Leukemia and Diffuse Large B-cell Lymphoma, run by Novartis Pharmaceuticals at 55 sites in 2 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 0 to 100 years
What is being tested?
CTL019
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
200 participants
Where
55 sitesin 2 countries
Study dates
21 Nov 2019to 31 Mar 2027
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 0 to 100 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

55 sites in 2 countries. Contact the study team or a site near you to ask about taking part.

Novartis Pharmaceuticals contact

+41613241111

Novartis.email@novartis.com

Novartis Pharmaceuticals contact

+81337978748

Novartis.email@novartis.com

55 sites in 2 countries

Japan49 sites, 49 recruiting

  • Novartis Investigative Site

    Aomori, 0308553

    Recruiting

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 1138431

    Recruiting

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 1138655

    Recruiting

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 1138677

    Recruiting

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo, 1138519

    Recruiting

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo, 1138603

    Recruiting

  • Novartis Investigative Site

    Chiba, 2608677

    Recruiting

  • Novartis Investigative Site

    Chuo Ku, Tokyo, 1040045

    Recruiting

  • Novartis Investigative Site

    Fuchū, Tokyo, 1838524

    Recruiting

  • Novartis Investigative Site

    Fuchū, Tokyo, 1838561

    Recruiting

  • Novartis Investigative Site

    Fukuoka, 8128582

    Recruiting

  • Novartis Investigative Site

    Gifu, Gifu, 5011194

    Recruiting

  • Novartis Investigative Site

    Hamamatsu, Shizuoka, 4313192

    Recruiting

  • Novartis Investigative Site

    Hiroshima, 7348551

    Recruiting

  • Novartis Investigative Site

    Ishikawa, 9208641

    Recruiting

  • Novartis Investigative Site

    Izumi, Osaka, 5941101

    Recruiting

  • Novartis Investigative Site

    Izumisano, Osaka, 5988577

    Recruiting

  • Novartis Investigative Site

    Izumo, Shimane, 6938501

    Recruiting

  • Novartis Investigative Site

    Kita-gun, Kagawa-ken, 7610793

    Recruiting

  • Novartis Investigative Site

    Kobe, Hyōgo, 6500047

    Recruiting

  • Novartis Investigative Site

    Kumamoto, Kumamoto, 8608556

    Recruiting

  • Novartis Investigative Site

    Kurashiki, Okayama-ken, 7108602

    Recruiting

  • Novartis Investigative Site

    Kyoto, Kyoto, 6028566

    Recruiting

  • Novartis Investigative Site

    Kyoto, 6068507

    Recruiting

  • Novartis Investigative Site

    Matsumoto, Nagano, 3908621

    Recruiting

  • Novartis Investigative Site

    Minato-ku, Tokyo, 1058471

    Recruiting

  • Novartis Investigative Site

    Nagasaki, Nagasaki, 8528501

    Recruiting

  • Novartis Investigative Site

    Nagoya, Aichi-ken, 4668560

    Recruiting

  • Novartis Investigative Site

    Nagoya, Aichi-ken, 4678602

    Recruiting

  • Novartis Investigative Site

    Niigata, 9518520

    Recruiting

  • Novartis Investigative Site

    Nishinomiya, Hyōgo, 6638501

    Recruiting

  • Novartis Investigative Site

    Okayama, 7008558

    Recruiting

  • Novartis Investigative Site

    Osaka, Osaka, 5418567

    Recruiting

  • Novartis Investigative Site

    Osaka, 5438555

    Recruiting

  • Novartis Investigative Site

    Osaka, 5458586

    Recruiting

  • Novartis Investigative Site

    Saitama, 3308777

    Recruiting

  • Novartis Investigative Site

    Sakai, Osaka, 5900197

    Recruiting

  • Novartis Investigative Site

    Sapporo, Hokkaido, 0608543

    Recruiting

  • Novartis Investigative Site

    Sapporo, Hokkaido, 0608648

    Recruiting

  • Novartis Investigative Site

    Sendai, Miyagi, 9808574

    Recruiting

  • Novartis Investigative Site

    Setagaya-ku, Tokyo, 1578535

    Recruiting

  • Novartis Investigative Site

    Shinjuku-ku, Tokyo, 1608582

    Recruiting

  • Novartis Investigative Site

    Suita, Osaka, 5650871

    Recruiting

  • Novartis Investigative Site

    Tōon, Ehime, 7910295

    Recruiting

  • Novartis Investigative Site

    Tsu, Mie-ken, 5148507

    Recruiting

  • Novartis Investigative Site

    Tsukuba, Ibaraki, 3058576

    Recruiting

  • Novartis Investigative Site

    Wakayama, 6418510

    Recruiting

  • Novartis Investigative Site

    Yokohama, Kanagawa, 2360004

    Recruiting

  • Novartis Investigative Site

    Yufu, Oita Prefecture, 8795593

    Recruiting

Other trials in Japan

Canada6 sites

  • Novartis Investigative Site

    Hamilton, Ontario, L8V 5C2

    Completed

  • Novartis Investigative Site

    Montreal, Quebec, H1T 2M4

    Withdrawn

  • Novartis Investigative Site

    Ottawa, Ontario, K1H 8L6

    Withdrawn

  • Novartis Investigative Site

    Québec, Quebec, G1R 2J6

    Withdrawn

  • Novartis Investigative Site

    Toronto, Ontario, M5G 1X8

    Completed

  • Novartis Investigative Site

    Toronto, Ontario, M5G 2M9

    Completed

Other trials in Canada

About this study

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

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.

Read the 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.

Being tested
CTL019
Keywords
out of specification, tisagenlecleucel, acute lymphoblastic leukemia, large B-cell lymphoma, relapsed/refractory, pediatric, young adult patients
Medical subject headings
Burkitt Lymphoma, Lymphoma, Large B-Cell, Diffuse, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CTL019Biological

    Also called Tisagenlecleucel

    A single intravenous (i.v.) infusion of CAR-positive viable T cells.

Study groups in NCT04094311
Study groupRoleWhat they receive
Group A: pALLExperimentalPediatric/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.
Receives: Biological: CTL019
Group B: r/r LBCLExperimentalAdult 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.
Receives: Biological: CTL019
Group C: r/r NHLExperimentalAdult 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.
Receives: Biological: CTL019

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of participants with Adverse Events (AEs)

    Percentage of participants with Serious AEs (SAEs) and non-SAEs

    Measured: From Screening up to 3 months for Part 1 and 1 day for Part 2

Secondary outcomes (2)

  1. Part 1: Overall Remission Rate in Group A (pALL)

    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).

    Measured: Up to 3 months

  2. Part 1: Overall Response Rate in Group B (LBCL)

    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).

    Measured: Up to 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Sep 2019
  2. Study start21 Nov 2019
  3. Primary completion31 Mar 2027 (estimated)
  4. Study completion31 Mar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
200 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04094311
Sponsor's protocol ID
CCTL019B2302
Status last verified
Sep 2026
First submitted
16 Sep 2019
Last updated
15 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing CTL019 in people with B-cell Acute Lymphoblastic Leukemia and Diffuse Large B-cell Lymphoma, run by Novartis Pharmaceuticals at 55 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 55 sites in Japan, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Novartis Pharmaceuticals (+41613241111, Novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of participants with Adverse Events (AEs) (time frame: From Screening up to 3 months for Part 1 and 1 day for Part 2). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04094311.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2026.

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