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Recruiting NCT04094311

Study of Out of Specification for Tisagenlecleucel

Phase III Interventional B-cell Acute Lymphoblastic Leukemia Diffuse Large B-cell Lymphoma

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: CTL019.
Who it may be relevant to
Registry conditions: B-cell Acute Lymphoblastic Leukemia, Diffuse Large B-cell Lymphoma. Basic parameters: 0 years — 100 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada, Japan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

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.

Interventions

  • Biological CTL019
    A single intravenous (i.v.) infusion of CAR-positive viable T cells.

Primary outcome measures

  • Percentage of participants with Adverse Events (AEs) [Time frame: From Screening up to 3 months for Part 1 and 1 day for Part 2]
Secondary outcome measures (2)
  • Part 1: Overall Remission Rate in Group A (pALL) [Time frame: Up to 3 months]
  • Part 1: Overall Response Rate in Group B (LBCL) [Time frame: Up to 3 months]

Eligibility criteria

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.

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Japan · 48 centers
  • Novartis Investigative Site — Nagoya
  • Novartis Investigative Site — Nagoya
  • Novartis Investigative Site — Tōon
  • Novartis Investigative Site — Gifu
  • Novartis Investigative Site — Sapporo
  • Novartis Investigative Site — Sapporo
  • Novartis Investigative Site — Kobe
  • Novartis Investigative Site — Nishinomiya
  • … and 40 more centers
Canada · 6 centers
  • Novartis Investigative Site — Hamilton
  • Novartis Investigative Site — Ottawa
  • Novartis Investigative Site — Toronto
  • Novartis Investigative Site — Toronto
  • Novartis Investigative Site — Montreal
  • Novartis Investigative Site — Québec

Identifiers

NCT: NCT04094311 · CCTL019B2302

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗