Clinical trial NCT05541341: Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies
Official title: Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies: a 15-year Prospective Registry Study on Three Cohorts.
Study type: OBSERVATIONAL
Overall status: RECRUITING
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
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-09-15
Enrollment: 200
Sex: ALL
Minimum age (years): 0
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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.

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

Detailed description: Eligible participants will be pediatric (\<18 years) and adult patients (aged 18 years or older) with B-cell malignancies who have received tisagenlecleucel through the commercial setting or out-of-specification (OOS) use in Brazil. We will collect data prospectively and complement missing information with retrospective data collection, when necessary. It is anticipated that approximately 200 patients will be enrolled in the cohort over 5 years divided among the study indications.

Since this is a non-interventional study, no administration of study drug or application of questionnaires will be mandated by this protocol. The study will consist of a "Pre-infusion" and a "Post infusion follow-up period" for up to 15 years post tisagenlecleucel infusion. All patients will be followed until death or last scheduled visit, whichever comes first.

For the study, "pre-infusion" and "follow-up post infusion" phases are defined as:

* "Pre-infusion" will consist of the patient's information from the time of diagnosis untiljust prior to infusion with tisagenlecleucel.
* "Follow-up Post infusion" information will comprise any information from the infusionof tisagenlecleucel onwards.

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.

Primary outcomes:
measure: Overall response rate (ORR)
timeFrame: Up to 15 years
measure: MRD negative overall response rate
timeFrame: Up to 15 years
measure: Duration of overall response (DOR)
timeFrame: Up to 15 years
measure: Relapse-free survival (RFS)
timeFrame: Up to 15 years
measure: Event-free survival (EFS) for ALL patients
timeFrame: Up to 15 years
measure: Progression free survival (PFS) for DLBCL patients
timeFrame: Up to 15 years
measure: Overall survival (OS)
timeFrame: Up to 15 years
measure: Number of ALL patients with hematologic recovery
timeFrame: Up to 15 years
Secondary outcomes:
measure: The type and frequency of SAEs and AE of special interest
timeFrame: Up to 15 years
measure: Incidence and severity of CRS and ICANS among HTLV 1 and 2 positive versus HTLV 1 and 2 negative patients
timeFrame: Up to 15 years
measure: Pregnancy rates
timeFrame: Up to 15 years
measure: Number of patients with confirmed secondary malignancies diagnosis
timeFrame: Up to 15 years
Other outcomes:
Locations:
city: Salvador
country: Brazil
facility: Novartis Investigative Site
state: BA
city: Minas Gerais
country: Brazil
facility: Novartis Investigative Site
state: Belo Horizonte
city: Curitiba
country: Brazil
facility: Novartis Investigative Site
state: PR
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: