Evaluating the effectiveness and safety of tisagenlecleucel therapy in Brazilian patients with B-cell malignancies

Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies: a 15-year Prospective Registry Study on Three Cohorts.

This study is testing how well and safely a treatment called tisagenlecleucel works for Brazilian patients with certain types of blood cancers, including lymphoma and leukemia, over a long follow-up period.

Quick facts

• Study type: Observational
• Enrollment: 200 (estimated)
• Ages: 0 Years to 100 Years
• Sex: All
• Sponsor: Novartis (Industry-sponsored)
• Locations: 7 sites (Minas Gerais, Belo Horizonte and 6 other locations)
• Trial ID: NCT05541341 on ClinicalTrials.gov

What this trial studies

This multicenter, national, non-interventional, prospective cohort study aims to assess the effectiveness and safety of tisagenlecleucel therapy in Brazilian patients diagnosed with B-lymphocyte malignancies, including Diffuse Large B-cell Lymphoma and Acute Lymphoblastic Leukemia. Eligible participants, both pediatric and adult, will have received tisagenlecleucel through commercial settings or out-of-specification use. The study will involve collecting data prospectively, with a follow-up period extending up to 15 years post-infusion, to monitor patient outcomes and safety. Approximately 200 patients are expected to be enrolled over a span of five years.

Who should consider this trial

Good fit: Ideal candidates include pediatric and adult patients with relapsed or refractory B-cell malignancies who have received tisagenlecleucel infusion.

Not a fit: Patients who did not consent to data collection or received tisagenlecleucel as part of an interventional clinical trial may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term effectiveness and safety of tisagenlecleucel therapy for patients with B-cell malignancies.

How similar studies have performed: Other studies have shown success with tisagenlecleucel therapy in similar patient populations, indicating a promising approach.

Eligibility criteria

Inclusion Criteria:

Patients eligible for inclusion in this study must meet the following criteria:

1. Patients who receive tisagenlecleucel infusion in the commercial setting or out-of-specification (OOS) use, AND
2. Signed informed consent must be obtained prior to participation in study, AND

   For ALL participants:
3. Patients of any gender aged 0-17 years (named as pediatric) with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
4. 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:
5. 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.

Exclusion Criteria:

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

Where this trial is running

Minas Gerais, Belo Horizonte and 6 other locations

• Novartis Investigative Site — Minas Gerais, Belo Horizonte, Brazil (Recruiting)
• Novartis Investigative Site — Salvador, Estado de Bahia, Brazil (Recruiting)
• Novartis Investigative Site — Curitiba, Paraná, Brazil (Recruiting)
• Novartis Investigative Site — São Paulo, São Paulo, Brazil (Recruiting)
• Novartis Investigative Site — São Paulo, São Paulo, Brazil (Recruiting)
• Novartis Investigative Site — São Paulo, Brazil (Recruiting)
• Novartis Investigative Site — São Paulo, Brazil (Recruiting)

Study contacts

• Study coordinator: Novartis Pharmaceuticals

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