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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 : September 16, 2021
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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 tisagenlecleucel 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, that is consistent with the Health Authority-approved indication in the product information for tisagenlecleucel in the respective country/region. In addition to safety, key efficacy of tisagenlecleucel that is out of specification (OOS) for commercial release will also be evaluated.


Condition or disease Intervention/treatment Phase
B-cell Acute Lymphoblastic Leukemia Diffuse Large B-cell Lymphoma Biological: Tisagenlecleucel Phase 3

Detailed Description:
Patients whose final manufactured tisagenlecleucel batches do not meet the approved local commercial release specifications are eligible for inclusion.Each case will be individually assessed for the expected benefit risk profile prior to release to the patient. Following the infusion of OOS tisagenlecleucel, the patient will be followed for 3 months on this study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 42 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Phase IIIB Study
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 : March 31, 2022
Estimated Study Completion Date : May 19, 2022


Arm Intervention/treatment
Tisagenlecleucel

All patients eligible for treatment with tisagenlecleucel per the Health Authority-approved tisagenlecleucel product information in the respective country/region are considered eligible for this study . Patients will be divided into 2 groups:

Group A: Pediatric and young adult patients with B-cell ALL (pALL) who meet the indication in Health Authority-approved tisagenlecleucel product information in the respective country/region whose final manufactured product is OOS for commercial release.

Group B: 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 product information for tisagenlecleucel in respective country/region but whose final manufactured product is OOS for commercial release/acceptance.

Biological: Tisagenlecleucel
A single intravenous (i.v.) infusion of CAR-positive viable T cells.
Other Name: CTL019




Primary Outcome Measures :
  1. Incidence of treatment emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths [ Time Frame: Screening through to EOT (Month 3)/Early Withdrawal ]
    Analysis of absolute and relative frequencies for treatment emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths by primary System Organ Class (SOC) to demonstrate the safety of OOS tisagenlecleucel in pediatric/young adult patients with relapsed/refractory B-cell Acute Lymphoblastic Leukemia (Group A, pALL) and Large B-cell Lymphoma (Group B, LBCL) through the monitoring of relevant clinical and laboratory safety parameters.


Secondary Outcome Measures :
  1. Group A (pALL): Overall Remission Rate (ORR) [ Time Frame: Screening through to EOT (Month 3)/Early Withdrawal ]
    ORR

  2. Group B (LBCL): Overall Response Rate [ Time Frame: Screening through to EOT (Month 3)/Early Withdrawal ]
    ORR



Information from the National Library of Medicine

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

  • 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.

Information from the National Library of Medicine

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


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 Novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +81337978748

Locations
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Canada, Ontario
Novartis Investigative Site Recruiting
Hamilton, Ontario, Canada, L8V 5C2
Novartis Investigative Site Recruiting
Toronto, Ontario, Canada, M5G 1X8
Novartis Investigative Site Recruiting
Toronto, Ontario, Canada, M5G 2M9
Canada
Novartis Investigative Site Recruiting
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
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
Yokohama-city, Kanagawa, Japan, 236-0004
Novartis Investigative Site Recruiting
Kyoto-city, Kyoto, Japan, 602-8566
Novartis Investigative Site Recruiting
Sendai city, Miyagi, Japan, 980 8574
Novartis Investigative Site Recruiting
Matsumoto-city, Nagano, Japan, 390-8621
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
Suita city, Osaka, Japan, 565 0871
Novartis Investigative Site Recruiting
Hamamatsu-city, Shizuoka, Japan, 431-3192
Novartis Investigative Site Recruiting
Bunkyo ku, Tokyo, Japan, 113-8431
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
Kyoto, Japan, 606 8507
Novartis Investigative Site Recruiting
Osaka, Japan, 545-8586
Novartis Investigative Site Recruiting
Saitama, Japan, 330 8777
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04094311    
Other Study ID Numbers: CCTL019B2302
First Posted: September 18, 2019    Key Record Dates
Last Update Posted: September 16, 2021
Last Verified: September 2021
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


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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
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
out of specification
tisagenlecleucel
acute lymphoblastic leukemia
large B-cell lymphoma
relapsed/refractory
pediatric
young adult patients
Additional relevant MeSH terms:
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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