Study of Out of Specification for Tisagenlecleucel

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

ClinicalTrials.gov Identifier: NCT04094311

Novartis Reference Number: CCTL019B2302

Last Update: Mar 06, 2023

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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.

Condition

B-cell Acute Lymphoblastic Leukemia

Diffuse Large B-cell Lymphoma

Phase

Phase 3

Overall status

Recruiting

Start date

Nov 21, 2019

Completion date

Dec 29, 2023

Gender

All

Age(s)

(Child, Adult, Older Adult)

Interventions

Biological

CTL019

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

Eligibility 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:

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.