Study of efficacy and safety of reinfusion of tisagenlecleucel in pediatric and young adult patients with Acute Lymphoblastic Leukemia

Phase 1

• Conditions: Paediatric patients with B-cell acute lymphoblastic leukaemia; MedDRA version: 21.0Level: LLTClassification code 10000845Term: Acute lymphoblastic leukemiaSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2021-001535-99-Outside-EU/EEA
• Lead Sponsor: ovartis Pharmaceuticals Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-03-14
• Last Update Posted: 21 March 2022

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

- Signed informed consent must be obtained prior to participation in the study
- Must have an additional dose of tisagenlecleucel available and prescribed by a physician in the course of medical practice
- Age up to and including 25 years
- Patients must have CD-19+ Leukemia
- Patients who were previously treated with tisagenlecleucel and present with evidence of B-cell recovery as defined by: Peripheral blood (PB) absolute B lymphocyte count = 50/µL, OR PB B lymphocyte = 10% of the total lymphocytes
Other protocol-defined Inclusion/Exclusion may apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 47
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 7
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-Prior gene therapy other than tisagenlecleucel
- Prior adoptive T cell therapy other than tisagenlecleucel
- Active CNS involvement by malignancy
- Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at screening
- HIV positive test within 8 weeks of screening
Other protocol-defined Inclusion/Exclusion may apply.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Evaluate the incidence of B cell aplasia after reinfusion of tisagenlecleucel;Secondary Objective: - Evaluate the efficacy of reinfusion of tisagenlecleucel for loss of B cell aplasia as measured by overall remission rate (ORR) 12 months after tisagenlecleucel reinfusion, which includes CR and CR with incomplete blood count recovery (CRi) as determined by investigator assessment for pALL patients<br>- Evaluate event free survival (EFS), Overall survival (OS) and the safety of reinfusion of tisagenlecleucel for B cell recovery or MRD.;Primary end point(s): Percentage of patients who establish B cell aplasia within 12 months of reinfusion, as measured by circulating B lymphocytes (< 50/µL) and presence of CTL019 cells by qPCR in the peripheral blood;Timepoint(s) of evaluation of this end point: reinfusion up to 12 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Percentage of patients with overall remission rate (ORR= CR + CRi) per Investigator assessment<br>-Event free survival (EFS)<br>- Overall Survival (OS) ;Timepoint(s) of evaluation of this end point: reinfusion up to 12 months