NCT04094311

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P25-P50 for phase_3

Timeline
11mo left

Started Nov 2019

Longer than P75 for phase_3

Geographic Reach
2 countries

53 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress88%
Nov 2019Mar 2027

First Submitted

Initial submission to the registry

September 16, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 18, 2019

Completed
2 months until next milestone

Study Start

First participant enrolled

November 21, 2019

Completed
7.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2027

Last Updated

April 20, 2026

Status Verified

April 1, 2026

Enrollment Period

7.4 years

First QC Date

September 16, 2019

Last Update Submit

April 17, 2026

Conditions

B-cell Acute Lymphoblastic LeukemiaDiffuse Large B-cell Lymphoma

Keywords

out of specificationtisagenlecleucelacute lymphoblastic leukemialarge B-cell lymphomarelapsed/refractorypediatricyoung adult patients

Outcome Measures

Primary Outcomes (1)

  • Percentage of participants with Adverse Events (AEs)

    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 Outcomes (2)

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

    Up to 3 months

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

    Up to 3 months

Study Arms (3)

Group A: pALL

EXPERIMENTAL

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

Group B: r/r LBCL

EXPERIMENTAL

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

Group C: r/r NHL

EXPERIMENTAL

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

Interventions

CTL019BIOLOGICAL

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

Also known as: Tisagenlecleucel
Group A: pALLGroup B: r/r LBCLGroup C: r/r NHL

Eligibility Criteria

Age0 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (53)

Novartis Investigative Site

Hamilton, Ontario, L8V 5C2, Canada

COMPLETED

Novartis Investigative Site

Ottawa, Ontario, K1H 8L6, Canada

WITHDRAWN

Novartis Investigative Site

Toronto, Ontario, M5G 1X8, Canada

COMPLETED

Novartis Investigative Site

Toronto, Ontario, M5G 2M9, Canada

COMPLETED

Novartis Investigative Site

Montreal, Quebec, H1T 2M4, Canada

WITHDRAWN

Novartis Investigative Site

Québec, Quebec, G1R 2J6, Canada

WITHDRAWN

Novartis Investigative Site

Nagoya, Aichi-ken, 4668560, Japan

RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, 4678602, Japan

RECRUITING

Novartis Investigative Site

Tōon, Ehime, 7910295, Japan

RECRUITING

Novartis Investigative Site

Gifu, Gifu, 501-1194, Japan

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 060-8648, Japan

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608543, Japan

RECRUITING

Novartis Investigative Site

Kobe, Hyōgo, 6500047, Japan

RECRUITING

Novartis Investigative Site

Nishinomiya, Hyōgo, 6638501, Japan

RECRUITING

Novartis Investigative Site

Tsukuba, Ibaraki, 3058576, Japan

RECRUITING

Novartis Investigative Site

Kita-gun, Kagawa-ken, 7610793, Japan

RECRUITING

Novartis Investigative Site

Yokohama, Kanagawa-ku, 236-0004, Japan

RECRUITING

Novartis Investigative Site

Kyoto, Kyoto, 602-8566, Japan

RECRUITING

Novartis Investigative Site

Tsu, Mie-ken, 514-8507, Japan

RECRUITING

Novartis Investigative Site

Sendai, Miyagi, 9808574, Japan

RECRUITING

Novartis Investigative Site

Matsumoto, Nagano, 3908621, Japan

RECRUITING

Novartis Investigative Site

Nagasaki, Nagasaki, 852-8501, Japan

RECRUITING

Novartis Investigative Site

Yufu, Oita Prefecture, 8795593, Japan

RECRUITING

Novartis Investigative Site

Kurashiki, Okayama-ken, 7108602, Japan

RECRUITING

Novartis Investigative Site

Izumi, Osaka, 5941101, Japan

RECRUITING

Novartis Investigative Site

Izumisano, Osaka, 5988577, Japan

RECRUITING

Novartis Investigative Site

Osaka, Osaka, 5418567, Japan

RECRUITING

Novartis Investigative Site

Sakai, Osaka, 590-0197, Japan

RECRUITING

Novartis Investigative Site

Suita, Osaka, 565-0871, Japan

RECRUITING

Novartis Investigative Site

Izumo, Shimane, 6938501, Japan

RECRUITING

Novartis Investigative Site

Hamamatsu, Shizuoka, 4313192, Japan

RECRUITING

Novartis Investigative Site

Bunkyo Ku, Tokyo, 113-8655, Japan

RECRUITING

Novartis Investigative Site

Bunkyo Ku, Tokyo, 1138431, Japan

RECRUITING

Novartis Investigative Site

Bunkyo Ku, Tokyo, 1138677, Japan

RECRUITING

Novartis Investigative Site

Bunkyo-ku, Tokyo, 1138519, Japan

RECRUITING

Novartis Investigative Site

Chuo Ku, Tokyo, 1040045, Japan

RECRUITING

Novartis Investigative Site

Fuchū, Tokyo, 1838524, Japan

RECRUITING

Novartis Investigative Site

Minato-ku, Tokyo, 105-8471, Japan

RECRUITING

Novartis Investigative Site

Setagaya-ku, Tokyo, 1578535, Japan

RECRUITING

Novartis Investigative Site

Shinjuku-ku, Tokyo, 1608582, Japan

RECRUITING

Novartis Investigative Site

Aomori, 0308553, Japan

RECRUITING

Novartis Investigative Site

Chiba, 2608677, Japan

RECRUITING

Novartis Investigative Site

Fukuoka, 8128582, Japan

RECRUITING

Novartis Investigative Site

Hiroshima, 7348551, Japan

RECRUITING

Novartis Investigative Site

Ishikawa, 9208641, Japan

RECRUITING

Novartis Investigative Site

Kumamoto, 860-8556, Japan

RECRUITING

Novartis Investigative Site

Kyoto, 6068507, Japan

RECRUITING

Novartis Investigative Site

Niigata, 9518520, Japan

RECRUITING

Novartis Investigative Site

Okayama, 7008558, Japan

RECRUITING

Novartis Investigative Site

Osaka, 543-8555, Japan

RECRUITING

Novartis Investigative Site

Osaka, 5458586, Japan

RECRUITING

Novartis Investigative Site

Saitama, 330-8777, Japan

RECRUITING

Novartis Investigative Site

Wakayama, 641-8510, Japan

RECRUITING

MeSH Terms

Conditions

Burkitt LymphomaLymphoma, Large B-Cell, DiffusePrecursor Cell Lymphoblastic Leukemia-LymphomaRecurrence

Interventions

tisagenlecleucel

Condition Hierarchy (Ancestors)

Epstein-Barr Virus InfectionsHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsTumor Virus InfectionsLymphoma, B-CellLymphoma, Non-HodgkinLymphomaNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, LymphoidLeukemiaHematologic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111Novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+81337978748Novartis.email@novartis.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 16, 2019

First Posted

September 18, 2019

Study Start

November 21, 2019

Primary Completion (Estimated)

March 31, 2027

Study Completion (Estimated)

March 31, 2027

Last Updated

April 20, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

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

Locations

JP(47)CA(6)