Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies

NCT05541341 | Recruiting

• 1 other identifier: CCTL019BBR02
• Study Type: observational
• Target: 200
• Locations: 1 country
• Sites: 7

Brief Summary

This will be a multicenter, national, non-interventional, prospective cohort study

Conditions

Diffuse Large B-cell Lymphoma; Acute Lymphoblastic Leukemia; Follicular Lymphoma

Keywords

Diffuse Large B-cell Lymphoma; DLBCL; Acute Lymphoblastic Leukemia; ALL; Tisagenlecleucel; Brazil; NIS; Follicular Lymphoma

Outcome Measures

Primary Outcomes (8)

• Overall response rate (ORR)

  The overall response rate will be defined as the total proportion of participants exhibiting either the best overall response (BOR) of complete or partial responses and the proportion of patient with BOR of CR/PR or CR/CRi for ALL patients will be reported along with its 95% CI. For ALL participants, the BOR will be defined as a CR or a CRi in accordance with National Comprehensive Cancer Network (NCCN) guidelines and previous guidelines (Appelbaum et al 2007)(Cheson et al 2003). For lymphomas, the BOR will be defined as a CR or PR in accordance with the Cheson response criteria (Cheson et al 2007) and the Lugano classification (Cheson et al 2016).

  Up to 15 years

• MRD negative overall response rate

  The percentage of B-cell ALL patients who achieve a Best Overall Response (BOR) of CR or CRi with a Minimal residual disease (MRD) negative bone marrow will be provided with 95% CI.

  Up to 15 years

• Duration of overall response (DOR)

  Duration of overall response (DOR) applies only to patients whose best overall disease response was either: * CR or PR for patients with lymphomas, or * CR or a CRi for patients with ALL. DOR will be defined as the time from the date of first documented disease response (Complete Response (CR) or PR for patients with lymphomas, and Complete Remission (CR) or CRi for patients with ALL), whichever occurs first, to the date of first documented progression or first documented relapse according to indication, or to the date of death due to the underlying disease. In case a patient does not have progression/relapse or death due to underlying disease (defined as the event for this outcome) prior to data cut-off, DOR will be censored at the date of the last assessment on or prior to the earliest censoring event.

  Up to 15 years

• Relapse-free survival (RFS)

  RFS is measured by the time from date of first documented disease response as CR or CRi to relapse or death due to any cause in ALL patients. In case a patient does not have relapse or death due to any cause prior to data cutoff, RFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.

  Up to 15 years

• Event-free survival (EFS) for ALL patients

  EFS is the time from date of first tisagenlecleucel infusion to treatment failure, relapse or death from any cause, whichever occurred first, for B-cell ALL patients.

  Up to 15 years

• Progression free survival (PFS) for DLBCL patients

  PFS is defined as the time from the date of first infusion to the date of event defined as the first documented progression of lymphoma or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of the last adequate assessment. In case a patient does not have progression or death prior to data cutoff, PFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.

  Up to 15 years

• Overall survival (OS)

  Overall survival is the time from date of first tisagenlecleucel infusion to the date of death due to any reason, In case a patient is alive at the date of last contact on or before data cutoff, OS is censored at the date of last contact.

  Up to 15 years

• Number of ALL patients with hematologic recovery

  Dates of hematological recovery (i.e., dates of Absolute Neutrophil Count (ANC) and platelet recovery) will be collected. ANC recovery is defined as an ANC of ≥ 0.5 × 109/L (500/mm\^3) for 3 consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of 3 consecutive laboratory values where the ANC is ≥ 0.5 × 109/L (CIBMTR). The first date of the 3 consecutive laboratory values obtained on different days where the platelet count was ≥ 20 × 109/L should be recorded. It should be ensured that no platelet transfusions were administered for 7 days immediately preceding this date (CIBMTR).

  Up to 15 years

Secondary Outcomes (4)

• The type and frequency of SAEs and AE of special interest

  Up to 15 years

• Incidence and severity of CRS and ICANS among HTLV 1 and 2 positive versus HTLV 1 and 2 negative patients

  Up to 15 years

• Pregnancy rates

  Up to 15 years

• Number of patients with confirmed secondary malignancies diagnosis

  Up to 15 years

Study Arms (3)

Acute Lymphoblastic Leukemia (ALL)

Children/young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia who received tisagenlecleucel infusion

Other: tisagenlecleucel

Diffuse Large B-cell Lymphoma (DLBCL)

Adult patients with relapsed/refractory Diffuse Large B-cell Lymphoma who received tisagenlecleucel infusion

Other: tisagenlecleucel

Follicular Lymphoma (FL)

Patients of any gender aged 18 year or older, with relapsed/refractory Follicular Lymphoma who received tisagenlecleucel infusion.

Other: tisagenlecleucel

Interventions

tisagenlecleucel OTHER

Prospective observational study. There is no treatment allocation. Patients prescribed with tisagenlecleucel in the commercial setting or out-of-specification (OOS) are eligible to enroll into this study

Acute Lymphoblastic Leukemia (ALL); Diffuse Large B-cell Lymphoma (DLBCL); Follicular Lymphoma (FL)

Eligibility Criteria

• Age: 0 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include pediatric/ adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia aged 0-25 years and adult patients aged ≥ 18 years with relapsed/refractory Diffuse Large B-cell Lymphoma and relapsed/refractory follicular lymphoma, who received tisagenlecleucel infusion in the commercial setting or out-of-specification.

You may qualify if:

• Patients who receive tisagenlecleucel infusion in the commercial setting or out-of-specification (OOS) use, AND
• Signed informed consent must be obtained prior to participation in study, AND
• For ALL participants:
• Patients of any gender aged 0-17 years (named as pediatric) with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
• Patients of any gender, aged 18-25 years (named as adults) - with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
• For DBLCL and FL participants:
• Patients of any gender aged 18 years or older, who have been diagnosed with relapsed/ refractory Diffuse Large B-cell Lymphoma and received tisagenlecleucel infusion.

You may not qualify if:

• Patients who did not consent to data collection.
• Patients who received tisagenlecleucel infusion as part of any interventional clinical trial.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (7)

Novartis Investigative Site

Minas Gerais, Belo Horizonte, 34006-059, Brazil

RECRUITING

Novartis Investigative Site

Salvador, Estado de Bahia, 41253-190, Brazil

RECRUITING

Novartis Investigative Site

Curitiba, Paraná, 81520-060, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 01323-900, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 04544-000, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, 01409-902, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, 01509-010, Brazil

RECRUITING

MeSH Terms

Conditions

Lymphoma, Large B-Cell, Diffuse; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Lymphoma, Follicular

Interventions

tisagenlecleucel

Condition Hierarchy (Ancestors)

Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Lymphoid; Leukemia; Hematologic Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

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

Study Record Dates

• First Submitted: September 13, 2022
• First Posted: September 15, 2022
• Study Start: November 23, 2023
• Primary Completion (Estimated): November 23, 2043
• Study Completion (Estimated): November 23, 2043
• Last Updated: June 6, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

BR (7)