A Real-world Study of the Effectiveness of Tisagenlecleucel in Acute Lymphoblastic Leukemia Patients

NCT07039383 | Completed

• 1 other identifier: CCTL019BUS08
• Study Type: observational
• Target: 79
• Locations: 1 country
• Sites: 1

Brief Summary

This was a retrospective, cross-sectional, center-based chart review study that collected real-world data for relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL) patients receiving tisagenlecleucel (tisa-cel). Five centers in the United States were included for the study. The date of the initial tisa-cel infusion was defined as the index date. A baseline period from ALL diagnosis to the index date was used to capture patient characteristics including demographics, and disease and treatment history. The study period, defined as the period from tisa-cel infusion to the end of follow-up date, i.e. the last contact date on medical charts or death, whichever was earlier, was used to capture the clinical outcomes of tisa-cel among ALL patients.

Conditions

Acute Lymphoblastic Leukemia

Keywords

Tisagenlecleucel; Real-world; Chart review

Outcome Measures

Primary Outcomes (1)

• Remission Rate

  Remission rate was defined as the best overall response as either complete remission (CR) or complete remission with incomplete blood count recovery (CRi) to tisa-cel. CR: \<5% blasts in bone marrow based on morphologic assessment, no evidence of extramedullary disease, full recovery of peripheral blood counts (platelets \> 100x10\^9/L, absolute neutrophil \> 1.0x10\^9/L, and circulating blasts \<1%), and blood transfusions independency (i.e., no transfusion or transfusion ≤7 days). CRi: \<5% blasts in bone marrow, no evidence of extramedullary disease, but without full recovery of peripheral blood counts with or without blood transfusions independency.

  Month 1 and Month 3

Secondary Outcomes (43)

• Number of Patients per Demographic Category

  Baseline

• Demgraphics: Weight

  Baseline

• Number of Patients by Disease Characteristics Category

  Baseline

• Number of Patients by Treatment Characteristics Category

  Baseline

• Lymphoblast Proportion in Bone Marrow Before Tisa-cel Infusion

  Baseline

Study Arms (1)

Tisa-cel Cohort

Patients with R/R B-cell ALL who received tisa-cel infusions between December 2020 and September 2022.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

You may qualify if:

• Diagnosed with B-cell ALL.
• Infusion of tisa-cel happened since December 2020 and at least 1 year before the chart abstraction date.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2025
• First Posted: June 26, 2025
• Study Start: March 1, 2023
• Primary Completion: June 18, 2024
• Study Completion: June 18, 2024
• Last Updated: June 26, 2025

Record last verified: 2025-06

Locations

US (1)