Clinical Trials /

Study of Out of Specification for Tisagenlecleucel

NCT04094311

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 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.

Related Conditions:
  • B-Cell Acute Lymphoblastic Leukemia
  • Diffuse Large B-Cell Lymphoma
  • High Grade B-Cell Lymphoma, Not Otherwise Specified
  • Transformed Non-Hodgkin Lymphoma
Recruiting Status:

Recruiting

Phase:

Phase 3

Trial Eligibility

Document

Title

  • Brief Title: Study of Out of Specification for Tisagenlecleucel
  • 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

Clinical Trial IDs

  • ORG STUDY ID: CCTL019B2302
  • NCT ID: NCT04094311

Conditions

  • B-cell Acute Lymphoblastic Leukemia
  • Diffuse Large B-cell Lymphoma

Interventions

DrugSynonymsArms
TisagenlecleucelCTL019Tisagenlecleucel

Purpose

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.

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.
    

Trial Arms

NameTypeDescriptionInterventions
TisagenlecleucelOtherAll 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.
  • Tisagenlecleucel

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:

          -  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.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Incidence of treatment emergent Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths
Time Frame:Screening through to EOT (Month 3)/Early Withdrawal
Safety Issue:
Description: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

Measure:Group A (pALL): Overall Remission Rate (ORR)
Time Frame:Screening through to EOT (Month 3)/Early Withdrawal
Safety Issue:
Description:ORR
Measure:Group B (LBCL): Overall Response Rate
Time Frame:Screening through to EOT (Month 3)/Early Withdrawal
Safety Issue:
Description:ORR

Details

Phase:Phase 3
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Novartis Pharmaceuticals

Trial Keywords

  • out of specification
  • tisagenlecleucel
  • acute lymphoblastic leukemia
  • large B-cell lymphoma
  • relapsed/refractory
  • pediatric
  • young adult patients

Last Updated

February 26, 2021